ES2134775T3 - Lipidos cationicos para entrega intracelular de moleculas biologicamente activas. - Google Patents

Lipidos cationicos para entrega intracelular de moleculas biologicamente activas.

Info

Publication number
ES2134775T3
ES2134775T3 ES91908905T ES91908905T ES2134775T3 ES 2134775 T3 ES2134775 T3 ES 2134775T3 ES 91908905 T ES91908905 T ES 91908905T ES 91908905 T ES91908905 T ES 91908905T ES 2134775 T3 ES2134775 T3 ES 2134775T3
Authority
ES
Spain
Prior art keywords
cationic lipids
biologically active
disclosed
cationic
active molecules
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Lifetime
Application number
ES91908905T
Other languages
English (en)
Inventor
Philip L Felgner
Raj Kumar
Channa Basava
Richard C Border
Jiin-Yu Hwang-Felgner
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Fresh Tracks Therapeutics Inc
Original Assignee
Vical Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Vical Inc filed Critical Vical Inc
Application granted granted Critical
Publication of ES2134775T3 publication Critical patent/ES2134775T3/es
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/186—Quaternary ammonium compounds, e.g. benzalkonium chloride or cetrimide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A61K47/544—Phospholipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0014—Skin, i.e. galenical aspects of topical compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Dermatology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Dispersion Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Virology (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)

Abstract

SE DESCRIBEN LIPIDOS CATIONICOS CAPACES DE FACILITAR EL TRANSPORTE DE AGENTES BIOLOGICAMENTE ACTIVOS A LAS CELULAS, QUE INCLUYE LA TRANSFECCION DE LAS CELULAS POR POLINUCLEOTIDOS TERAPEUTICOS, LA DISTRIBUCION DE MEDICAMENTOS ANTIVIRICOS Y LA INTRODUCCION DE PEPTIDOS INMUNOGENICOS. LOS LIPIDOS CATIONICOS, QUE COMPRENDEN UN GRUPO AMONIO, TIENEN LA ESTRUCTURA GENERAL (I). TAMBIEN SE DESCRIBEN ADUCTOS DE ESTOS COMPUESTOS QUE COMPRENDEN PUNTOS CATIONICOS ADICIONALES QUE INCREMENTAN LA ACTIVIDAD TRANSFECTIVA O DE TRANSPORTE. LAS CORRELACIONES ESTRUCTURAACTIVIDAD MANTIENEN LA SELECCION DE COMPUESTOS PREFERIDOS A SINTETIZAR CON ESTE FIN. LAS COMPOSICIONES DESCRITAS PARA USO DE ESTOS LIPIDOS CATIONICOS INCLUYEN LAS FORMULACIONES PARA LA TRANSFECCION IN VITRO Y LAS FORMULACIONES FARMACEUTICAS PARA LA ADMINISTRACION PARENTERAL Y TOPICA DE LOS AGENTES TERAPEUTICOS.
ES91908905T 1990-04-19 1991-04-18 Lipidos cationicos para entrega intracelular de moleculas biologicamente activas. Expired - Lifetime ES2134775T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US51121990A 1990-04-19 1990-04-19
US56344490A 1990-08-07 1990-08-07
US07/686,746 US5264618A (en) 1990-04-19 1991-04-16 Cationic lipids for intracellular delivery of biologically active molecules

Publications (1)

Publication Number Publication Date
ES2134775T3 true ES2134775T3 (es) 1999-10-16

Family

ID=27414476

Family Applications (1)

Application Number Title Priority Date Filing Date
ES91908905T Expired - Lifetime ES2134775T3 (es) 1990-04-19 1991-04-18 Lipidos cationicos para entrega intracelular de moleculas biologicamente activas.

Country Status (11)

Country Link
US (2) US5264618A (es)
EP (1) EP0523189B1 (es)
JP (1) JP2538474B2 (es)
AT (1) ATE181319T1 (es)
AU (1) AU7854791A (es)
CA (1) CA2079814C (es)
DE (1) DE69131347T2 (es)
DK (1) DK0523189T3 (es)
ES (1) ES2134775T3 (es)
GR (1) GR3030974T3 (es)
WO (1) WO1991016024A1 (es)

Families Citing this family (1493)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE145337T1 (de) * 1988-05-02 1996-12-15 Phanos Tech Inc Verbindungen, zusammensetzungen und verfahren zum binden von bio-affektions-substanzen an oberflächenmembranen von bioteilchen
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US6214804B1 (en) 1989-03-21 2001-04-10 Vical Incorporated Induction of a protective immune response in a mammal by injecting a DNA sequence
US6867195B1 (en) 1989-03-21 2005-03-15 Vical Incorporated Lipid-mediated polynucleotide administration to reduce likelihood of subject's becoming infected
ATE165516T1 (de) * 1989-03-21 1998-05-15 Vical Inc Expression von exogenen polynukleotidsequenzen in wirbeltieren
US6706694B1 (en) 1990-03-21 2004-03-16 Vical Incorporated Expression of exogenous polynucleotide sequences in a vertebrate
US6228844B1 (en) 1991-11-12 2001-05-08 Vical Incorporated Stimulating vascular growth by administration of DNA sequences encoding VEGF
US5543389A (en) * 1990-11-01 1996-08-06 State Of Oregon, Acting By And Through The Oregon State Board Of Higher Education On Behalf Of The Oregon Health Sciences University, A Non Profit Organization Covalent polar lipid-peptide conjugates for use in salves
US5543390A (en) * 1990-11-01 1996-08-06 State Of Oregon, Acting By And Through The Oregon State Board Of Higher Education, Acting For And On Behalf Of The Oregon Health Sciences University Covalent microparticle-drug conjugates for biological targeting
US5256641A (en) * 1990-11-01 1993-10-26 State Of Oregon Covalent polar lipid-peptide conjugates for immunological targeting
US5827819A (en) * 1990-11-01 1998-10-27 Oregon Health Sciences University Covalent polar lipid conjugates with neurologically active compounds for targeting
US6605712B1 (en) * 1990-12-20 2003-08-12 Arch Development Corporation Gene transcription and ionizing radiation: methods and compositions
US6156304A (en) * 1990-12-20 2000-12-05 University Of Pittsburgh Of The Commonwealth System Of Higher Education Gene transfer for studying and treating a connective tissue of a mammalian host
US7202225B1 (en) * 1993-10-15 2007-04-10 Emanuele R Martin Therapeutic delivery compositions and methods of use thereof
US20020123476A1 (en) * 1991-03-19 2002-09-05 Emanuele R. Martin Therapeutic delivery compositions and methods of use thereof
US5693769A (en) * 1991-12-13 1997-12-02 Transcell Technologies, Inc. Glycosylated steroid derivatives for transport across biological membranes and process for making and using same
US5795870A (en) * 1991-12-13 1998-08-18 Trustees Of Princeton University Compositions and methods for cell transformation
AU3467193A (en) 1991-12-17 1993-07-19 Regents Of The University Of California, The Gene therapy for cystic fibrosis transmembrane conductance regulator activity (CFTR)
US5756353A (en) * 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
US5858784A (en) * 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
US6627615B1 (en) 1991-12-17 2003-09-30 The Regents Of The University Of California Methods and compositions for in vivo gene therapy
CA2128616A1 (en) * 1992-01-23 1993-08-05 Gary H. Rhodes Ex vivo gene transfer
US5885970A (en) * 1992-03-16 1999-03-23 Isis Pharmaceuticals, Inc. Antisense oligonucleotides against human protein kinase C
US6113946A (en) * 1992-04-03 2000-09-05 The Regents Of The University Of California Self-assembling polynucleotide delivery system comprising dendrimer polycations
CA2133323C (en) * 1992-04-03 2010-10-26 Francis C. Szoka, Jr. Self-assembling polynucleotide delivery system
US5792751A (en) * 1992-04-13 1998-08-11 Baylor College Of Medicine Tranformation of cells associated with fluid spaces
US20020108136A1 (en) * 1997-03-21 2002-08-08 Sri Transgenic animals produced by homologous sequence targeting
US6806084B1 (en) * 1992-06-04 2004-10-19 The Regents Of The University Of California Methods for compositions for in vivo gene delivery
EP0646178A1 (en) * 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
EP0607408A4 (en) * 1992-07-13 1997-12-10 Eukarion Inc Transvascular and intracellular delivery of lipidized proteins.
US7105574B1 (en) 1999-03-26 2006-09-12 Vical Incorporated Adjuvant compositions and methods for enhancing immune responses to polynucleotide-based vaccines
US5334761A (en) * 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
IL107150A0 (en) * 1992-09-29 1993-12-28 Isis Pharmaceuticals Inc Oligonucleotides having a conserved g4 core sequence
US5329029A (en) * 1992-11-05 1994-07-12 Wan Barbara Y Phosphatidylalkanolamine derivatives and their use in generating phospholipid conjugates
US5552155A (en) * 1992-12-04 1996-09-03 The Liposome Company, Inc. Fusogenic lipsomes and methods for making and using same
US5705655A (en) * 1992-12-17 1998-01-06 Megabios Corporation Amphiphilic nitrogen containing imidazolinium derivative compounds and uses
US5631237A (en) * 1992-12-22 1997-05-20 Dzau; Victor J. Method for producing in vivo delivery of therapeutic agents via liposomes
US6090925A (en) 1993-03-09 2000-07-18 Epic Therapeutics, Inc. Macromolecular microparticles and methods of production and use
ES2113094T3 (es) * 1993-03-09 1998-04-16 Epic Therapeutics Inc Las microparticulas macromoleculares y metodos de obtencion.
EP1624068A1 (en) * 1993-06-01 2006-02-08 Life Technologies Inc. Genetic immunization with cationic lipids
WO1995002698A1 (en) * 1993-07-12 1995-01-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5563111A (en) * 1993-08-03 1996-10-08 Kao Corporation Agricultural chemical composition comprising amine surfactants with at least one ester or amide linkage
US6348449B1 (en) * 1993-09-21 2002-02-19 The Trustees Of The University Of Pennsylvania Methods of inducing mucosal immunity
WO1995014381A1 (en) * 1993-11-24 1995-06-01 Megabios Corporation Amphiphilic derivatives of guanidine
US5665879A (en) * 1993-11-24 1997-09-09 Megabios Corporation Amphiphilic derivatives of piperazine
NZ277614A (en) * 1993-11-24 1998-05-27 Megabios Corp Piperazine-containing amphiphiles and their use in liposomes for delivering genetic material intracellularly
US5674908A (en) * 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
US6613351B1 (en) * 1993-12-20 2003-09-02 Biotech Tools S.A. Compound capable of introducing at least one molecule into a cell
US6057299A (en) * 1994-01-13 2000-05-02 Calydon, Inc. Tissue-specific enhancer active in prostate
US5830686A (en) * 1994-01-13 1998-11-03 Calydon Tissue-specific enhancer active in prostate
US5928944A (en) * 1994-02-04 1999-07-27 The United States Of America As Represented By The Department Of Health And Human Services Method of adenoviral-medicated cell transfection
US6989434B1 (en) 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
US6075012A (en) * 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
US5942496A (en) 1994-02-18 1999-08-24 The Regent Of The University Of Michigan Methods and compositions for multiple gene transfer into bone cells
US5962427A (en) 1994-02-18 1999-10-05 The Regent Of The University Of Michigan In vivo gene transfer methods for wound healing
US5763416A (en) 1994-02-18 1998-06-09 The Regent Of The University Of Michigan Gene transfer into bone cells and tissues
US6074840A (en) 1994-02-18 2000-06-13 The Regents Of The University Of Michigan Recombinant production of latent TGF-beta binding protein-3 (LTBP-3)
US6995008B1 (en) 1994-03-07 2006-02-07 Merck & Co., Inc. Coordinate in vivo gene expression
US6551618B2 (en) 1994-03-15 2003-04-22 University Of Birmingham Compositions and methods for delivery of agents for neuronal regeneration and survival
US5651981A (en) * 1994-03-29 1997-07-29 Northwestern University Cationic phospholipids for transfection
US5739118A (en) 1994-04-01 1998-04-14 Apollon, Inc. Compositions and methods for delivery of genetic material
EP0759768A4 (en) * 1994-04-07 2000-08-09 Proteinix Company VASOACTIVE COLON PEPTIDE
AU2589095A (en) * 1994-05-16 1995-12-05 Washington University Cell membrane fusion composition and method
JPH10506093A (ja) * 1994-06-22 1998-06-16 メガバイオス・コーポレイション カチオン性両親媒性化合物
US5777153A (en) * 1994-07-08 1998-07-07 Gilead Sciences, Inc. Cationic lipids
FR2722506B1 (fr) * 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
DE4426429A1 (de) * 1994-07-26 1996-02-01 Boehringer Ingelheim Int Verfahren zum Einführen von DNA in höhere eukaryotische Zellen
US7592317B1 (en) * 1994-08-11 2009-09-22 The University Of Chicago Constitutive gene expression in conjuction with ionizing radiation
AUPM747694A0 (en) * 1994-08-16 1994-09-08 Commonwealth Scientific And Industrial Research Organisation Delivery of nucleic acids and peptides
US5527928A (en) * 1994-09-30 1996-06-18 Nantz; Michael H. Cationic transport reagents
US5892071A (en) * 1994-09-30 1999-04-06 The Reagents Of The University Of California Cationic transport reagents
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
DE69527206T2 (de) * 1994-09-30 2003-02-27 Inex Pharmaceuticals Corp., Vancouver Mittel zum einbringen polyanionischer materialien in zellen
US5753613A (en) * 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US6911216B1 (en) 1994-10-12 2005-06-28 Genzyme Corporation Targeted delivery via biodegradable polymers
US5641665A (en) * 1994-11-28 1997-06-24 Vical Incorporated Plasmids suitable for IL-2 expression
US5840710A (en) * 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
US5948767A (en) * 1994-12-09 1999-09-07 Genzyme Corporation Cationic amphiphile/DNA complexes
US5719131A (en) * 1994-12-09 1998-02-17 Genzyme Corporation Cationic amphiphiles containing dialkylamine lipophilic groups for intracellular delivery of therapeutic molecules
US5747471A (en) * 1994-12-09 1998-05-05 Genzyme Corporation Cationic amphiphiles containing steroid lipophilic groups for intracellular delivery of therapeutic molecules
US5767099A (en) * 1994-12-09 1998-06-16 Genzyme Corporation Cationic amphiphiles containing amino acid or dervatized amino acid groups for intracellular delivery of therapeutic molecules
US5939401A (en) * 1994-12-09 1999-08-17 Genzyme Corporation Cationic amphiphile compositions for intracellular delivery of therapeutic molecules
US5910487A (en) * 1994-12-09 1999-06-08 Genzyme Corporation Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules
US6383814B1 (en) 1994-12-09 2002-05-07 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US6071890A (en) * 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
US5650096A (en) * 1994-12-09 1997-07-22 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US6331524B1 (en) 1994-12-09 2001-12-18 Genzyme Corporation Organ-specific targeting of cationic amphiphile / DNA complexes for gene therapy
US6630455B1 (en) 1995-01-13 2003-10-07 Vanderbilt University Methods for inducing mucosal immune responses
US6008202A (en) * 1995-01-23 1999-12-28 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5929043A (en) 1995-01-31 1999-07-27 Pangene Corporation Recombinase mediated DNA therapies
US5830430A (en) 1995-02-21 1998-11-03 Imarx Pharmaceutical Corp. Cationic lipids and the use thereof
CN100569297C (zh) 1995-02-28 2009-12-16 加利福尼亚大学董事会 基因转移介导的血管形成疗法
US20030148968A1 (en) * 1995-02-28 2003-08-07 Hammond H. Kirk Techniques and compositions for treating cardiovascular disease by in vivo gene delivery
US6752987B1 (en) 1995-02-28 2004-06-22 The Regents Of The University Of California Adenovirus encoding human adenylylcyclase (AC) VI
US5962428A (en) * 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
US20040043971A1 (en) * 1995-04-03 2004-03-04 Bone Care International, Inc. Method of treating and preventing hyperparathyroidism with active vitamin D analogs
US6242434B1 (en) * 1997-08-08 2001-06-05 Bone Care International, Inc. 24-hydroxyvitamin D, analogs and uses thereof
US20020183288A1 (en) * 1995-04-03 2002-12-05 Bone Care International, Inc. Method for treating and preventing hyperparathyroidism
CA2214029A1 (en) * 1995-04-25 1996-10-31 Magda Marquet Single-vial formulations of dna/lipid complexes
US6420549B1 (en) 1995-06-06 2002-07-16 Isis Pharmaceuticals, Inc. Oligonucleotide analogs having modified dimers
US5711964A (en) * 1995-06-07 1998-01-27 United States Of America Method for the intracellular delivery of biomolecules using liposomes containing cationic lipids and vitamin D
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US5705385A (en) * 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5994315A (en) * 1995-06-07 1999-11-30 East Carolina University Low adenosine agent, composition, kit and method for treatment of airway disease
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5851548A (en) * 1995-06-07 1998-12-22 Gen-Probe Incorporated Liposomes containing cationic lipids and vitamin D
JPH11507031A (ja) * 1995-06-07 1999-06-22 ジェン−プローブ・インコーポレーテッド チオカチオン性脂質，医薬組成物およびその使用方法
US5739271A (en) * 1995-06-07 1998-04-14 Gen-Probe Incorporated Thiocationic lipids
CA2223179A1 (en) 1995-06-07 1996-12-19 Bob Dale Brown Phosphonic acid-based cationic lipids
WO1996040961A1 (en) * 1995-06-07 1996-12-19 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US5756352A (en) * 1995-06-07 1998-05-26 Gen-Probe Incorporated Thiocationic lipid-nucleic acid conjugates
US5869040A (en) * 1995-06-07 1999-02-09 Biogen, Inc Gene therapy methods and compositions
US5759519A (en) * 1995-06-07 1998-06-02 Gen-Probe Incorporated Method for the intracellular delivery of biomolecules using thiocationic lipids
JPH11507352A (ja) 1995-06-07 1999-06-29 ジンタ・インコーポレイテッド 新規カルバメート基本カチオン性脂質
US20030069173A1 (en) * 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
CA2222328C (en) * 1995-06-07 2012-01-10 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
DE19521412A1 (de) * 1995-06-14 1996-12-19 Boehringer Mannheim Gmbh Neue kationische und polykationische Amphiphile, diese enthaltende Reagenzien und deren Verwendung
US5931809A (en) * 1995-07-14 1999-08-03 Depotech Corporation Epidural administration of therapeutic compounds with sustained rate of release
US6339173B1 (en) 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
JPH11510489A (ja) * 1995-07-21 1999-09-14 ジンタ・インコーポレイテッド 新規アミド基本カチオン性脂質
US5855911A (en) * 1995-08-29 1999-01-05 Board Of Regents, The University Of Texas System Liposomal phosphodiester, phosphorothioate, and P-ethoxy oligonucleotides
NO953680D0 (no) * 1995-09-18 1995-09-18 Hans Prydz Cellesyklusenzymer
US6120794A (en) * 1995-09-26 2000-09-19 University Of Pittsburgh Emulsion and micellar formulations for the delivery of biologically active substances to cells
AU7078396A (en) * 1995-09-27 1997-04-30 Regents Of The University Of California, The Polyfunctional cationic cytofectins, formulations and methods for generating active cytofectin:polynucleotide transfection complexes
US5869715A (en) * 1995-09-27 1999-02-09 The Reagents Of The University Of California Polyfunctional cationic cytofectins
US6086913A (en) * 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
EP0863749A2 (en) * 1995-11-30 1998-09-16 Vical Incorporated Complex cationic lipids
US6537776B1 (en) 1999-06-14 2003-03-25 Diversa Corporation Synthetic ligation reassembly in directed evolution
US6126964A (en) * 1996-01-04 2000-10-03 Mirus Corporation Process of making a compound by forming a polymer from a template drug
AUPN741696A0 (en) * 1996-01-05 1996-01-25 Commonwealth Scientific And Industrial Research Organisation Delivery of nucleic acids ii
US6392069B2 (en) * 1996-01-08 2002-05-21 Canji, Inc. Compositions for enhancing delivery of nucleic acids to cells
US7002027B1 (en) * 1996-01-08 2006-02-21 Canji, Inc. Compositions and methods for therapeutic use
US20040014709A1 (en) * 1996-01-08 2004-01-22 Canji, Inc. Methods and compositions for interferon therapy
US5789244A (en) * 1996-01-08 1998-08-04 Canji, Inc. Compositions and methods for the treatment of cancer using recombinant viral vector delivery systems
DE69736385T2 (de) * 1996-02-09 2007-08-23 Pi-Wan Cheng Kationisches lipid und rezeptorligand erleichterte gabe von biologisch aktiven molekuelen
WO1997034483A1 (en) * 1996-03-18 1997-09-25 California Institute Of Technology Methods for increasing or decreasing transfection efficiency
US5783566A (en) * 1996-05-10 1998-07-21 California Institute Of Technology Method for increasing or decreasing transfection efficiency
US5994317A (en) * 1996-04-09 1999-11-30 Vical Incorporated Quaternary cytofectins
US5798250A (en) * 1996-04-15 1998-08-25 Icn Pharmaceuticals, Inc. Facilitating hepatitus virus infection in cell culture using a membrane stressor
CA2252617A1 (en) * 1996-05-01 1997-11-06 Imarx Pharmaceutical Corp. Methods for delivering compounds into a cell
SK152698A3 (en) * 1996-05-08 1999-05-07 Nika Health Products Ltd Cationic virosomes as transfer system for genetic material
WO1997042819A1 (en) * 1996-05-13 1997-11-20 Alberto Haces Cationic lipids for transfection of negatively charged or neutral molecules into living cells
US5980935A (en) * 1996-05-15 1999-11-09 Kirpotin; Dmitri Cationic lipids and methods of use therefor
US6274136B1 (en) * 1996-05-29 2001-08-14 University Of Southern California Construction and use of genes encoding pathogenic epitopes for treatment of autoimmune disease
US8323963B2 (en) * 1996-05-29 2012-12-04 University Of Southern California Construction and use of genes encoding pathogenic epitopes for treatment of autoimmune disease
US5935936A (en) * 1996-06-03 1999-08-10 Genzyme Corporation Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US6093816A (en) * 1996-06-27 2000-07-25 Isis Pharmaceuticals, Inc. Cationic lipids
WO1998000554A1 (en) * 1996-07-03 1998-01-08 Genetics Institute, Inc. Protease fmh-1, an ice/ced-like protease
CA2261887C (en) * 1996-07-23 2007-10-02 Oregon Health Sciences University Covalent polar lipid conjugates with biologically-active compounds for use in salves
CA2271388C (en) * 1996-09-13 2007-11-06 The School Of Pharmacy, University Of London Liposomes encapsulating polynucleotides operatively coding for immunogenic polypeptides
US7384923B2 (en) * 1999-05-14 2008-06-10 Lipoxen Technologies Limited Liposomes
GB2321642B8 (en) 1996-10-01 2006-08-22 Geron Corp Human telomerase reverse transcriptase promoter
US5861397A (en) * 1996-10-03 1999-01-19 Vical Incorporated Piperazine based cytofectins
US6977244B2 (en) 1996-10-04 2005-12-20 Board Of Regents, The University Of Texas Systems Inhibition of Bcl-2 protein expression by liposomal antisense oligodeoxynucleotides
FR2754828B1 (fr) * 1996-10-23 1998-12-24 Univ Toulouse Structure membranaire artificielle, procede et polymere pour sa preparation, procede de preparation de ce polymere, particule et film comprenant cette structure
EP0956001B1 (en) * 1996-11-12 2012-09-12 The Regents of The University of California Preparation of stable formulations of lipid-nucleic acid complexes for efficient in vivo delivery
US6210707B1 (en) * 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
US5965441A (en) * 1996-11-13 1999-10-12 The General Hospital Coporation HSV/AAV hybrid amplicon vectors
EP0958380A4 (en) * 1996-12-06 2004-03-17 Univ California MACROMOLECUL LIPID COMPLEX AND METHOD FOR THE PRODUCTION AND USE THEREOF
CN1167793C (zh) 1996-12-06 2004-09-22 阿温蒂斯药物公司 人脂酶样基因编码的多肽及利用它的组合物和方法
US7008776B1 (en) * 1996-12-06 2006-03-07 Aventis Pharmaceuticals Inc. Compositions and methods for effecting the levels of high density lipoprotein (HDL) cholesterol and apolipoprotein AI very low density lipoprotein (VLDL) cholesterol and low density lipoprotein (LDL) cholesterol
US20020128240A1 (en) * 1996-12-30 2002-09-12 Bone Care International, Inc. Treatment of hyperproliferative diseases using active vitamin D analogues
US6527716B1 (en) 1997-12-30 2003-03-04 Altea Technologies, Inc. Microporation of tissue for delivery of bioactive agents
JP2001511811A (ja) * 1997-02-13 2001-08-14 ボーン ケア インターナショナル インコーポレイテッド ビタミンｄ化合物の標的治療放出
US20030129194A1 (en) * 1997-02-13 2003-07-10 Bone Care International, Inc. Targeted therapeutic delivery of vitamin D compounds
FR2760193B1 (fr) * 1997-02-28 1999-05-28 Transgene Sa Lipides et complexes de lipides cationiques et de substances actives, notamment pour la transfection de cellules
US7112338B2 (en) * 1997-03-12 2006-09-26 The Regents Of The University Of California Cationic liposome delivery of taxanes to angiogenic blood vessels
US5837283A (en) * 1997-03-12 1998-11-17 The Regents Of The University Of California Cationic lipid compositions targeting angiogenic endothelial cells
US5965542A (en) * 1997-03-18 1999-10-12 Inex Pharmaceuticals Corp. Use of temperature to control the size of cationic liposome/plasmid DNA complexes
US5948653A (en) * 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination
US5925628A (en) * 1997-03-31 1999-07-20 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US6235310B1 (en) 1997-04-04 2001-05-22 Valentis, Inc. Methods of delivery using cationic lipids and helper lipids
US5958894A (en) * 1997-04-04 1999-09-28 Megabios Corporation Amphiphilic biguanide derivatives
US5912239A (en) * 1997-04-04 1999-06-15 Genzyme Corporation Imidazole-containing cationic amphiphiles for intracellular delivery of therapeutic molecules
FR2761912B1 (fr) * 1997-04-14 1999-07-02 Capsulis Procede destine a faire adherer un produit sur une surface
JP2001523103A (ja) 1997-04-28 2001-11-20 ローヌ−プーラン・ロレ・エス・アー 腫瘍治療用血管形成アンタゴニストのアデノウイルスによる腫瘍内送達
EP0979311A1 (en) * 1997-04-30 2000-02-16 Of The University Of Minnesota Regents $i(IN VIVO) USE OF RECOMBINAGENIC OLIGONUCLEOBASES TO CORRECT GENETIC LESIONS IN HEPATOCYTES
US5948925A (en) * 1997-05-06 1999-09-07 Genzyme Corporation Cationic amphiphiles containing linkers derived from neutral or positively charged amino acids
US5952516A (en) * 1997-05-08 1999-09-14 Genzyme Corporation Cationic amphiphiles containing multiplesteroid lipophilic groups
US5942634A (en) * 1997-05-09 1999-08-24 Genzyme Corporation Cationic amphiphiles for cell transfections
US20030104044A1 (en) * 1997-05-14 2003-06-05 Semple Sean C. Compositions for stimulating cytokine secretion and inducing an immune response
EP1027033B1 (en) 1997-05-14 2009-07-22 The University Of British Columbia High efficiency encapsulation of nucleic acids in lipid vesicles
US6835395B1 (en) 1997-05-14 2004-12-28 The University Of British Columbia Composition containing small multilamellar oligodeoxynucleotide-containing lipid vesicles
WO1999002192A1 (en) * 1997-07-11 1999-01-21 Brandeis University Method of inducing apoptosis by reducing the level of thiamin
US20090239940A1 (en) * 1997-07-22 2009-09-24 Del Monte Federica Treating heart failure and ventricular arrhythmias
JP4302877B2 (ja) 1997-07-30 2009-07-29 エモリー・ユニバーシテイ 新規な骨鉱化蛋白質、ｄｎａ、ベクター及び発現系
US7923250B2 (en) 1997-07-30 2011-04-12 Warsaw Orthopedic, Inc. Methods of expressing LIM mineralization protein in non-osseous cells
EP1002103B1 (en) 1997-08-04 2007-10-17 Cell Genesys, Inc. A human glandular kallikrein enhancer, vectors comprising the enhancer and methods of use thereof
DE69840216D1 (de) * 1997-08-08 2008-12-24 Celmed Oncology Usa Inc Verfahren und Zubereitungen um Resistenz gegen biologische oder chemische Therapien zu Überwinden
US6281371B1 (en) 1997-08-13 2001-08-28 Biontex Laboratories Gmbh Lipopolyamines, and the preparation and use thereof
US6818627B1 (en) 1997-08-14 2004-11-16 The Trustees Of The University Of Pennsylvania Functional fragments of HIV-1 Vpr protein and methods of using the same
US7285288B1 (en) 1997-10-03 2007-10-23 Board Of Regents, The University Of Texas System Inhibition of Bcl-2 protein expression by liposomal antisense oligodeoxynucleotides
US7704962B1 (en) 1997-10-03 2010-04-27 Board Of Regents, The University Of Texas System Small oligonucleotides with anti-tumor activity
US6884842B2 (en) 1997-10-14 2005-04-26 Alnis Biosciences, Inc. Molecular compounds having complementary surfaces to targets
US6875606B1 (en) * 1997-10-23 2005-04-05 The United States Of America As Represented By The Department Of Veterans Affairs Human α-7 nicotinic receptor promoter
US5998482A (en) * 1997-11-10 1999-12-07 David; Sunil A. Use of synthetic polycationic amphiphilic substances with fatty acid or hydrocarbon substituents as anti-sepsis agents
EP1987845B1 (en) 1997-11-20 2012-03-21 Vical Incorporated Treatment of cancer using cytokine-expressing polynucleotides and compositions therefor.
DE69829995T2 (de) * 1997-12-01 2006-02-23 Fang, Fang, San Diego Multivalente rekombinante antikörper zur behandlung von hrv infektionen
US7964192B1 (en) 1997-12-02 2011-06-21 Janssen Alzheimer Immunotherapy Prevention and treatment of amyloidgenic disease
US7179892B2 (en) 2000-12-06 2007-02-20 Neuralab Limited Humanized antibodies that recognize beta amyloid peptide
TWI239847B (en) 1997-12-02 2005-09-21 Elan Pharm Inc N-terminal fragment of Abeta peptide and an adjuvant for preventing and treating amyloidogenic disease
FR2772047B1 (fr) 1997-12-05 2004-04-09 Ct Nat D Etudes Veterinaires E Sequence genomique et polypeptides de circovirus associe a la maladie de l'amaigrissement du porcelet (map), applications au diagnostic et a la prevention et/ou au traitement de l'infection
US20010003580A1 (en) 1998-01-14 2001-06-14 Poh K. Hui Preparation of a lipid blend and a phospholipid suspension containing the lipid blend
WO1999036514A1 (en) * 1998-01-16 1999-07-22 California Pacific Medical Center Research Institute Methods and compositions for gene delivery
TWI244924B (en) 1998-01-23 2005-12-11 Newbiotics Inc Enzyme catalyzed therapeutic agents
US7462605B2 (en) * 1998-01-23 2008-12-09 Celmed Oncology (Usa), Inc. Phosphoramidate compounds and methods of use
US6410328B1 (en) * 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
JP2002506625A (ja) 1998-03-19 2002-03-05 ヒューマン ジノーム サイエンシーズ， インコーポレイテッド サイトカインレセプター共通γ鎖様
US6271209B1 (en) * 1998-04-03 2001-08-07 Valentis, Inc. Cationic lipid formulation delivering nucleic acid to peritoneal tumors
US6043390A (en) 1998-04-03 2000-03-28 The Regents Of The University Of California Pentaerythritol lipid derivatives and nucleic-acid complexes
EP1075519B1 (en) 1998-05-06 2005-11-09 Vlaams Interuniversitair Instituut voor Biotechnologie vzw. Inhibitors of nf-kb activation
US6225456B1 (en) 1998-05-07 2001-05-01 University Technololy Corporation Ras suppressor SUR-5
US6506889B1 (en) 1998-05-19 2003-01-14 University Technology Corporation Ras suppressor SUR-8 and related compositions and methods
BR9910787A (pt) * 1998-05-29 2001-10-30 Us Health Métodos e composições úteis para modulação deangiogênese usando tirosina cinase src
US6200598B1 (en) * 1998-06-18 2001-03-13 Duke University Temperature-sensitive liposomal formulation
US6726925B1 (en) 1998-06-18 2004-04-27 Duke University Temperature-sensitive liposomal formulation
US20040077603A1 (en) * 1998-08-28 2004-04-22 Wilhelm Stoffel Synthetic tear fluid
AU5911899A (en) * 1998-09-09 2000-03-27 Genzyme Corporation Methylation of plasmid vectors
US6135976A (en) 1998-09-25 2000-10-24 Ekos Corporation Method, device and kit for performing gene therapy
US6077709A (en) 1998-09-29 2000-06-20 Isis Pharmaceuticals Inc. Antisense modulation of Survivin expression
NZ512244A (en) 1998-11-12 2003-12-19 Invitrogen Corp Polycationic transfection reagents for introducing anions into a cell
WO2000030444A1 (en) 1998-11-25 2000-06-02 Vanderbilt University Cationic liposomes for gene transfer
DE69918711T2 (de) * 1998-11-25 2005-07-21 Gene Therapy Systems, Inc., San Diego Amphiphile polyamin verbindungen
US20030035798A1 (en) * 2000-08-16 2003-02-20 Fang Fang Humanized antibodies
US6627215B1 (en) 1998-12-30 2003-09-30 Oligos Etc. Inc. Devices for improved wound management
US6441156B1 (en) * 1998-12-30 2002-08-27 The United States Of America As Represented By The Department Of Health And Human Services Calcium channel compositions and methods of use thereof
US20020032164A1 (en) 1998-12-30 2002-03-14 Dale Roderic M. K. Antimicrobial compounds and methods for their use
US7868162B2 (en) 1998-12-30 2011-01-11 Lakewood-Amedex, Inc. Antimicrobial and antiviral compounds and methods for their use
US6570003B1 (en) * 2001-01-09 2003-05-27 Lexion Genetics Incorporated Human 7TM proteins and polynucleotides encoding the same
US20020164784A1 (en) * 2000-10-27 2002-11-07 Walke D. Wade Novel human 7TM proteins and polynucleotides encoding the same
AU776150B2 (en) * 1999-01-28 2004-08-26 Medical College Of Georgia Research Institute, Inc. Composition and method for (in vivo) and (in vitro) attenuation of gene expression using double stranded RNA
US6750198B1 (en) 1999-02-25 2004-06-15 Fumedica Gmbh Preparation for the treatment of pigmentation disorders
KR20010103788A (ko) 1999-03-03 2001-11-23 멕코나시 에블린 에이치. 백신 및 유전자요법 조성물 및 이들의 제조 및 사용방법
US7393548B2 (en) * 1999-03-22 2008-07-01 J.P. M.E.D. Ltd. Nano oil in glycerin emulsion
AU4032300A (en) 1999-03-24 2000-10-09 Gene Therapy Systems, Inc. Method for generating transcriptionally active dna fragments
ATE272410T1 (de) 1999-03-26 2004-08-15 Vical Inc Adjuvanszusammensetzungen zur erhöhung der immunantwort auf polynukleotid-basierende impfstoffe
US6379698B1 (en) 1999-04-06 2002-04-30 Isis Pharmaceuticals, Inc. Fusogenic lipids and vesicles
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US6696424B1 (en) 1999-05-28 2004-02-24 Vical Incorporated Cytofectin dimers and methods of use thereof
CA2375727C (en) * 1999-05-28 2010-05-25 Vical Incorporated Cytofectin dimers and methods of use thereof
UA81216C2 (en) 1999-06-01 2007-12-25 Prevention and treatment of amyloid disease
US6943152B1 (en) 1999-06-10 2005-09-13 Merial DNA vaccine-PCV
US20010048940A1 (en) * 1999-06-18 2001-12-06 Jennifer D. Tousignant Cationic amphiphile micellar complexes
FR2797402B1 (fr) 1999-07-15 2004-03-12 Biomerieux Stelhys Utilisation d'un polypeptide pour detecter, prevenir ou traiter un etat pathologique associe a une maladie degenerative, neurologique ou autoimmune
IL147749A0 (en) 1999-07-22 2002-08-14 Newbiotics Inc Methods for treating therapy-resistant tumors
US6683061B1 (en) * 1999-07-22 2004-01-27 Newbiotics, Inc. Enzyme catalyzed therapeutic activation
US20030078499A1 (en) * 1999-08-12 2003-04-24 Eppstein Jonathan A. Microporation of tissue for delivery of bioactive agents
US20050249794A1 (en) * 1999-08-27 2005-11-10 Semple Sean C Compositions for stimulating cytokine secretion and inducing an immune response
AUPQ259399A0 (en) * 1999-09-01 1999-09-23 Lustre Investments Pte Ltd Therapeutic agents
US20020115838A1 (en) * 2000-08-22 2002-08-22 Friddle Carl Johan Novel human proteases and polynucleotides encoding the same
US20050153323A1 (en) * 2000-07-28 2005-07-14 Yi Hu Novel human proteases and polynucleotides encoding the same
US6716614B1 (en) 1999-09-02 2004-04-06 Lexicon Genetics Incorporated Human calcium dependent proteases, polynucleotides encoding the same, and uses thereof
US6448388B1 (en) 2000-08-16 2002-09-10 Lexicon Genetics Incorporated Human proteases and polynucleotides encoding the same
US20080003673A1 (en) * 1999-09-02 2008-01-03 Alejandro Abuin Novel human proteases and polynucleotides encoding the same
US20030050464A1 (en) * 2000-07-28 2003-03-13 Yi Hu Novel human proteases and polynucleotides encoding the same
HUP0202669A3 (en) * 1999-09-09 2004-06-28 Univ California Cationic liposome delivery of taxanes to angiogenic bloos vessels
US6867291B1 (en) * 2000-09-15 2005-03-15 Lexicon Genetics Incorporated Human hemicentin proteins and polynucleotides encoding the same
US6790660B1 (en) * 2001-09-18 2004-09-14 Lexicon Genetics Incorporated Human kielin-like proteins and polynucleotides encoding the same
US6720173B1 (en) 1999-11-08 2004-04-13 Lexicon Genetics Incorporated Human kinase protein and polynucleotides encoding the same
US6476210B2 (en) 2000-10-12 2002-11-05 Lexicon Genetics Incorporated Human kinases and polynucleotides encoding the same
US6797510B1 (en) * 2001-05-24 2004-09-28 Lexicon Genetics Incorporated Human kinases and polynucleotides encoding the same
US6586230B1 (en) 2000-10-27 2003-07-01 Lexicon Genetics Incorporated Human kinase and polynucleotides encoding the same
US6511840B1 (en) 2000-06-15 2003-01-28 Lexicon Genetics Incorporated Human kinase proteins and polynucleotides encoding the same
US6746861B2 (en) 2000-01-18 2004-06-08 Lexicon Genetics Incorporated Human kinase protein and polynucleotides encoding the same
US6593125B2 (en) 2000-11-20 2003-07-15 Lexicon Genetics Incorporated Human kinases and polynucleotides encoding the same
US6841377B1 (en) * 2001-06-13 2005-01-11 Lexicon Genetics Incorporated Human kinase and polynucleotides encoding the same
US20080050809A1 (en) * 1999-09-28 2008-02-28 Alejandro Abuin Novel human kinases and polynucleotides encoding the same
US6541252B1 (en) 2000-05-19 2003-04-01 Lexicon Genetics Incorporated Human kinases and polynucleotides encoding the same
US7892541B1 (en) 1999-09-30 2011-02-22 Tumor Biology Investment Group, Inc. Soluble epidermal growth factor receptor isoforms
US20080213878A1 (en) * 1999-10-19 2008-09-04 Gregory Donoho Novel human membrane proteins and polynucleotides encoding the same
EP1242588A2 (en) 1999-11-12 2002-09-25 Lexicon Genetics Incorporated Human proteins with homology to carboxypeptidases and polynucleotides encoding the same
AU1920601A (en) * 1999-11-19 2001-05-30 Lexicon Genetics Incorporated Novel human secreted proteins and polynucleotides encoding the same
ES2170622B1 (es) * 1999-12-03 2004-05-16 Consejo Superior De Investigaciones Cientificas Clones y vectores infectivos derivados de coronavirus y sus aplicaciones.
WO2001042287A2 (en) * 1999-12-07 2001-06-14 Lexicon Genetics Incorporated Putative human g-protein coupled receptors
JP2004504005A (ja) 1999-12-07 2004-02-12 レキシコン・ジェネティクス・インコーポレーテッド ヒト新規キナーゼタンパクおよびそれをコードするポリヌクレオチド
JP2003516154A (ja) * 1999-12-13 2003-05-13 レキシコン・ジェネティクス・インコーポレーテッド 新規ヒトトランスフェラーゼタンパク質及び該タンパク質をコードするポリヌクレオチド
US20030008813A1 (en) * 1999-12-17 2003-01-09 Felgner Philip L. Intracellular protein delivery compositions and methods of use
WO2001046417A2 (en) * 1999-12-22 2001-06-28 Lexicon Genetics Incorporated Human membrane proteins and polynucleotides encoding the same having homology to cd20 proteins and ige receptors
WO2001046407A1 (en) * 1999-12-22 2001-06-28 Lexicon Genetics Incorporated Polynucleotides encoding human protease homologs
FR2802925B1 (fr) * 1999-12-23 2002-03-22 Commissariat Energie Atomique Amphiphiles cationiques, leurs applications et leur procede de synthese
CA2395839A1 (en) * 1999-12-27 2001-07-05 The Regents Of The University Of California Gene therapy for congestive heart failure
EP1242609A2 (en) * 1999-12-30 2002-09-25 Novartis AG Novel colloid synthetic vectors for gene therapy
JP2004502402A (ja) * 2000-01-06 2004-01-29 レキシコン・ジェネティクス・インコーポレーテッド 新規ヒトプロテアーゼおよびそれをコードするポリヌクレオチド
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US20020055479A1 (en) 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
US20040002472A1 (en) * 2000-01-21 2004-01-01 Audonnet Jean-Christophe Francis Vaccination or immunization using a prime-boost regimen
US7078388B2 (en) * 2000-01-21 2006-07-18 Merial DNA vaccines for farm animals, in particular bovines and porcines
US6852705B2 (en) 2000-01-21 2005-02-08 Merial DNA vaccines for farm animals, in particular bovines and porcines
JP2004500084A (ja) * 2000-01-26 2004-01-08 レキシコン・ジェネティクス・インコーポレーテッド 新規ヒトニューレキシン様タンパク質及び該タンパク質をコードするポリヌクレオチド
EP1250433A1 (en) * 2000-01-28 2002-10-23 Lexicon Genetics Incorporated Human membrane proteins and polynucleotides encoding the same
EP1301600A2 (en) * 2000-01-28 2003-04-16 Lexicon Genetics Incorporated Human enzymes and polynucleotides encoding the same
EP1252317B1 (en) * 2000-02-04 2007-07-04 Lexicon Genetics Incorporated Human enzymes and polynucleotides encoding the same
EP1257579B1 (en) * 2000-02-04 2009-08-05 Lexicon Pharmaceuticals, Inc. Novel human g protein coupled receptor proteins and polynucleotides encoding the same
EP1254369B1 (en) 2000-02-08 2010-10-06 Sangamo BioSciences, Inc. Cells for drug discovery
EP1255843A1 (en) 2000-02-11 2002-11-13 Lexicon Genetics Incorporated Human proteases and polynucleotides encoding the same
CN1184076C (zh) 2000-02-16 2005-01-12 精工爱普生株式会社 喷墨打印机用墨盒
AU2001238503A1 (en) * 2000-02-17 2001-08-27 Lexicon Genetics Incorporated Novel human thrombospondin repeat proteins and polynucleotides encoding the same
US6555669B2 (en) 2000-02-29 2003-04-29 Lexicon Genetics Incorporated Human transferase proteins and polynucleotides encoding the same
WO2001064878A2 (en) * 2000-02-29 2001-09-07 Lexicon Genetics Incorporated Human transporter proteins and polynucleotides encoding the same
US20020082405A1 (en) * 2000-03-06 2002-06-27 Gregory Donoho Novel human transporter proteins and polynucleotides encoding the same
JP2004500106A (ja) 2000-03-10 2004-01-08 レキシコン・ジェネティクス・インコーポレーテッド 新規ヒトｇ共役タンパク質受容体キナーゼおよびそれをコードするポリヌクレオチド
CA2403205A1 (en) 2000-03-13 2001-09-20 Engene, Inc. Compositions and methods of regulated protein expression in gut endocrine k cells
EP1317551B1 (en) * 2000-03-13 2007-02-28 Lexicon Genetics Incorporated Human phospholipases and polynucleotides encoding the same
US7223395B2 (en) 2000-03-13 2007-05-29 Cornell Research Foundation, Inc. Blocking leukocyte emigration and inflammation by interfering with CD99/HEC2
AU2001245871B2 (en) * 2000-03-20 2006-06-01 Lexicon Pharmaceuticals, Inc. Novel human secreted proteins and polynucleotides encoding the same
US20040033600A1 (en) * 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
WO2001070763A1 (en) * 2000-03-22 2001-09-27 The Children's Hospital Of Philadelphia Modified blood clotting factors and methods of use
GB0018307D0 (en) 2000-07-26 2000-09-13 Aventis Pharm Prod Inc Polypeptides
EP1272634A1 (en) * 2000-04-03 2003-01-08 Lexicon Genetics Incorporated Human ion channel protein and polynucleotides encoding the same
CA2405557C (en) 2000-04-12 2013-09-24 Human Genome Sciences, Inc. Albumin fusion proteins
WO2001079509A1 (en) * 2000-04-12 2001-10-25 Lexicon Genetics Incorporated Human metalloprotease and polynucleotides encoding the same
AU5345901A (en) 2000-04-13 2001-10-30 Univ Rockefeller Enhancement of antibody-mediated immune responses
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
WO2001080897A2 (en) * 2000-04-21 2001-11-01 Vical Incorporated Compositions and methods for in vivo delivery of polynucleotide-based therapeutics
EP1276873A2 (en) 2000-04-25 2003-01-22 Lexicon Genetics Incorporated Human kinase proteins and polynucleotides encoding the same
US20020107375A1 (en) * 2000-05-12 2002-08-08 Brian Mathur Novel human lipocalin homologs and polynucleotides encoding the same
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
WO2001088133A2 (en) * 2000-05-18 2001-11-22 Lexicon Genetics Incorporated Human semaphorin homologs and polynucleotides encoding the same
WO2001090179A2 (en) * 2000-05-23 2001-11-29 Lexicon Genetics Incorporated Novel human thrombospondin-like proteins and polynucleotides encoding the same
US6790667B1 (en) * 2000-05-30 2004-09-14 Lexicon Genetics Incorporated Human mitochondrial proteins and polynucleotides encoding the same
EP2182005B1 (en) 2000-06-05 2015-03-25 The Brigham & Women's Hospital, Inc. A gene encoding a multidrug resistance human P-glycoprotein homologue on chromosome 7p15-21 and uses thereof
US6703495B2 (en) * 2000-06-07 2004-03-09 Lexicon Genetics Incorporated Polynucleotides encoding human transporter protein
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
AU2001264317A1 (en) * 2000-06-20 2002-01-02 Koken Co., Ltd. Preparations for oligonucleotide transfer
EP1297134A2 (en) * 2000-06-27 2003-04-02 Lexicon Genetics Incorporated Novel human gaba receptors and polynucleotides encoding the same
IL153378A0 (en) * 2000-07-18 2003-07-06 Bone Care Internat Inc STABILIZED 1alpha-HYDROXY VITAMIN D
AU2002224553A1 (en) * 2000-07-21 2002-02-05 Incyte Genomics, Inc. Human proteases
US6887685B1 (en) * 2000-07-25 2005-05-03 Lexicon Genetics Incorporated Human thymosin protein and polynucleotides encoding the same
ATE381351T1 (de) * 2000-07-27 2008-01-15 Univ Pennsylvania Anwendung des herpes simplex virus glykoproteins- d zur unterdrückung von immunantworten
US20020076780A1 (en) * 2000-08-16 2002-06-20 Turner C. Alexander Novel human ion channel proteins and polynucleotides encoding the same
JP2004506447A (ja) * 2000-08-22 2004-03-04 レキシコン・ジェネティクス・インコーポレーテッド 新規ヒト７ｔｍタンパクおよびそれをコードするポリヌクレオチド
AU2001288478B2 (en) 2000-08-25 2006-11-02 Basf Plant Science Gmbh Plant polynucleotides encoding prenyl proteases
AU2001285326A1 (en) * 2000-08-31 2002-03-13 Lexicon Genetics Incorporated Human kinase proteins and polynucleotides encoding the same
US7229790B2 (en) * 2000-09-01 2007-06-12 Lexicon Pharmaceuticals, Inc. Human GABA transporter protein and polynucleotides encoding the same
WO2002026981A2 (en) * 2000-09-27 2002-04-04 Lexicon Genetics Incorporated Human protease inhibitor proteins and polynucleotides encoding the same
ATE326533T1 (de) * 2000-09-27 2006-06-15 Lexicon Genetics Inc Menschliche ionenaustauschproteine und dafür kodierende polynukleotiden
US6777232B1 (en) 2000-10-02 2004-08-17 Lexicon Genetics Incorporated Human membrane proteins and polynucleotides encoding the same
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
US20050059031A1 (en) 2000-10-06 2005-03-17 Quantum Dot Corporation Method for enhancing transport of semiconductor nanocrystals across biological membranes
US7001763B1 (en) 2000-10-17 2006-02-21 Lexicon Genetics Incorporated Human semaphorin proteins and polynucleotides encoding the same
US6831059B2 (en) 2000-10-20 2004-12-14 Allergan, Inc. Compositions and methods for treating gonadotrophin related illnesses
AU2002232387A1 (en) * 2000-10-27 2002-05-06 Invitrogen Corporation Method for introducing antisense oligonucleotides into eucaryotic cells
US7060442B2 (en) * 2000-10-30 2006-06-13 Regents Of The University Of Michigan Modulators on Nod2 signaling
WO2002036633A2 (en) * 2000-10-30 2002-05-10 Lexicon Genetics Incorporated Human 7tm proteins and polynucleotides encoding the same
AU3087502A (en) * 2000-11-01 2002-05-15 Lexicon Genetics Inc Novel human proteases and polynucleotides encoding the same
CA2429519A1 (en) * 2000-11-15 2002-06-27 Lexicon Genetics Incorporated Novel human secreted proteins and polynucleotides encoding the same
CZ301427B6 (cs) * 2000-11-29 2010-02-24 Pci Biotech As Zpusob zavedení molekuly do cytosolu bunky
PE20020574A1 (es) 2000-12-06 2002-07-02 Wyeth Corp Anticuerpos humanizados que reconocen el peptido amiloideo beta
CA2431007A1 (en) 2000-12-11 2002-07-18 Lexicon Genetics Incorporated Novel human kinase and polynucleotides encoding the same
AU2002232642B2 (en) * 2000-12-12 2007-02-01 Lexicon Pharmaceuticals, Inc. Novel human kinases and uses thereof
US6583269B1 (en) * 2000-12-18 2003-06-24 Lexicon Genetics Incorporated Human protease inhibitor and polynucleotides encoding the same
JP2005502307A (ja) * 2000-12-20 2005-01-27 レキシコン・ジェネティクス・インコーポレーテッド 新規ヒトイオンチャンネルタンパクおよびそれをコードするポリヌクレオチド
US6852844B1 (en) 2000-12-20 2005-02-08 Lexicon Genetics Incorporated Human protocadherin proteins and polynucleotides encoding the same
WO2002059325A2 (en) * 2000-12-27 2002-08-01 Lexicon Genetics Incorporated Human kinases and polynucleotides encoding the same
DE10064870B4 (de) * 2000-12-27 2004-10-07 G.O.T. Gesellschaft Zur Therapieoptimierung Und Targeting Entwicklungs Mbh Schwefelhaltige Amphiphile für den Transfer von biologisch aktiven Molekülen in Zellen
DE60144198D1 (de) 2000-12-28 2011-04-21 Wyeth Llc Rekombinantes schutzprotein aus streptococcus pneumoniae
WO2002053753A2 (en) * 2001-01-05 2002-07-11 Lexicon Genetics Incorporated Novel human lipase and polynucleotides encoding the same
WO2002053754A2 (en) * 2001-01-08 2002-07-11 Lexicon Genetics Incorporated Human protease and polynucleotides encoding the same
EP1359921A4 (en) * 2001-01-19 2006-09-06 Newbiotics Inc METHOD FOR THE TREATMENT OF AUTOIMMUNE DISEASES AND INFLAMMATORY DISEASES
WO2002059287A2 (en) * 2001-01-23 2002-08-01 Lexicon Genetics Incorporated Novel human kinases and polynucleotides encoding the same
CA2434897A1 (en) * 2001-01-24 2002-08-01 Lexicon Genetics Incorporated Human lipase and polynucleotides encoding the same
EP1229134A3 (en) * 2001-01-31 2004-01-28 Nucleonics, Inc Use of post-transcriptional gene silencing for identifying nucleic acid sequences that modulate the function of a cell
US20020127674A1 (en) * 2001-02-02 2002-09-12 Xuanchuan Yu Novel human transporter protein and polynucleotides encoding the same
EP1373470B1 (en) * 2001-02-20 2013-04-24 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
WO2002066613A2 (en) 2001-02-20 2002-08-29 Rheogene Holdings, Inc Novel ecdysone receptor/invertebrate retinoid x receptor-based inducible gene expression system
AU2002247214B2 (en) * 2001-02-20 2008-04-03 Intrexon Corporation Chimeric retinoid X receptors and their use in a novel ecdysone receptor-based inducible gene expression system
CA2473197A1 (en) * 2001-02-20 2002-08-29 Lexicon Genetics Incorporated Novel human protease and polynucleotides encoding the same
US9249207B2 (en) 2001-02-20 2016-02-02 Intrexon Corporation Substitution mutant receptors and their use in an ecdysone receptor-based inducible gene expression system
ATE448793T1 (de) 2001-03-02 2009-12-15 Univ Rockefeller Rekombinante hybrid-allergenkonstrukte mit verringerter allergenität unter beibehaltung der immunogenität des natürlichen allergens
JP3929250B2 (ja) * 2001-03-08 2007-06-13 株式会社ルネサステクノロジ 半導体装置
WO2002072611A2 (en) * 2001-03-12 2002-09-19 Lexicon Genetics Incorporated Novel human egf-family proteins and polynucleotides encoding the same
AU2002245593A1 (en) * 2001-03-12 2002-09-24 Lexicon Genetics Incorporated Novel human dectin proteins and polynucleotides encoding the same
WO2002072774A2 (en) * 2001-03-12 2002-09-19 Lexicon Genetics Incorporated Novel human transporter proteins and polynucleotides encoding the same
AU2002254174A1 (en) * 2001-03-13 2002-09-24 Vical Incorporated Interferon-beta polynucleotide therapy for autoimmune and inflammatory diseases
US6994995B1 (en) * 2001-03-16 2006-02-07 Lexicon Genetics Incorporated Human synaptotagmin and polynucleotides encoding the same
US6613534B2 (en) 2001-03-20 2003-09-02 Wake Forest University Health Sciences MAP-2 as a determinant of metastatic potential
WO2002074932A2 (en) 2001-03-20 2002-09-26 Lexicon Genetics Incorporated Novel human kinase and polynucleotides encoding the same
US20030186390A1 (en) * 2001-03-22 2003-10-02 De Jong Gary Methods for delivering nucleic acid molecules into cells and assessment thereof
US7294511B2 (en) 2001-03-22 2007-11-13 Chromos Molecular Systems, Inc. Methods for delivering nucleic acid molecules into cells and assessment thereof
US6936469B2 (en) * 2001-03-22 2005-08-30 Chromos Molecular Systems Inc. Methods for delivering nucleic acid molecules into cells and assessment thereof
US20030096414A1 (en) * 2001-03-27 2003-05-22 Invitrogen Corporation Culture medium for cell growth and transfection
US20040028651A1 (en) * 2001-03-29 2004-02-12 Karrupiah Muthumani Composition and methods of using hiv vpr
US20040242523A1 (en) * 2003-03-06 2004-12-02 Ana-Farber Cancer Institue And The Univiersity Of Chicago Chemo-inducible cancer gene therapy
JP2004532213A (ja) * 2001-04-06 2004-10-21 ザ ユニヴァーシティ オヴ シカゴ Ｅｇｒ−１プロモーター活性の化学療法誘導
US8034791B2 (en) 2001-04-06 2011-10-11 The University Of Chicago Activation of Egr-1 promoter by DNA damaging chemotherapeutics
CA2443319A1 (en) * 2001-04-06 2002-10-17 Lexicon Genetics Incorporated Novel human kinase and polynucleotides encoding the same
CA2443516A1 (en) 2001-04-06 2002-10-17 Lexicon Genetics Incorporated Novel human kinases and polynucleotides encoding the same
US6644173B2 (en) * 2001-04-11 2003-11-11 Keuring, Incorporated Beverage filter cartridge holder
MXPA03009294A (es) * 2001-04-13 2004-04-20 Wyeth Corp Proteinas de superficie de streptococcus pyogenes.
US20070128229A1 (en) * 2002-04-12 2007-06-07 Wyeth Surface proteins of Streptococcus pyogenes
DK1385864T3 (da) 2001-04-13 2010-08-16 Human Genome Sciences Inc Anti-VEGF-2-antistoffer
WO2002083706A1 (en) 2001-04-16 2002-10-24 Lexicon Genetics Incorporated Nucleic acid encoding a human adenylosuccinate synthetase
US20020165175A1 (en) * 2001-04-17 2002-11-07 Xiaowu Liang Fast and enzymeless cloning of nucleic acid fragments
US20030077829A1 (en) * 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
AU2002259011A1 (en) * 2001-04-30 2002-11-11 Lexicon Genetics Incorporated Novel human nuclear transporters and polynucleotides encoding the same
CA2445947A1 (en) * 2001-04-30 2002-11-07 Targeted Genetics Corporation Lipid-comprising drug delivery complexes and methods for their production
WO2002090517A2 (en) * 2001-05-09 2002-11-14 Lexicon Genetics Incorporated Novel human kinases and polynucleotides encoding the same
US20020193585A1 (en) * 2001-05-25 2002-12-19 Walke D. Wade Novel human transporter proteins and polynucleotides encoding the same
DE60225064T2 (de) 2001-05-29 2009-01-29 Lexicon Pharmaceuticals, Inc., The Woodlands Neue menschliche hydroxylasen und diese codierende polynukleotide
US7745398B2 (en) * 2001-05-31 2010-06-29 Tumor Biology Investment Group, Inc. Soluble ErbB3 and treatment of cancer
US7744882B2 (en) 2001-05-31 2010-06-29 Tumor Biology Investment Group, Inc. Soluble ErbB3 methods of detection and antibodies
EP1406645A4 (en) * 2001-06-14 2005-02-16 Lexicon Genetics Inc NOVEL HUMAN TRANSPORTER PROTEINS AND POLYNUCLEOTIDES ENCODING THESE PROTEINS
US7803915B2 (en) 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
EP1407787B1 (en) * 2001-06-20 2009-04-29 Dainippon Sumitomo Pharma Co., Ltd. Method of promoting nucleic acid transfer
KR100628425B1 (ko) 2001-06-20 2006-09-28 제넨테크, 인크. 종양의 진단 및 치료를 위한 방법 및 이를 위한 조성물
CA2790034A1 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
DE60213596T2 (de) 2001-06-22 2007-09-13 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw. Abin zum schutz gegen hepatitis
JP2004536089A (ja) * 2001-06-26 2004-12-02 イッサム・リサーチ・ディベロップメント・カンパニー・オブ・ザ・ヘブルー・ユニバーシティ・オブ・エルサレム 物質を細胞内に導入するための配合物及び方法
WO2003004609A2 (en) * 2001-07-03 2003-01-16 Lexicon Genetics Incorporated Novel human kielin-like proteins and polynucleotides encoding the same
CN100344646C (zh) 2001-07-10 2007-10-24 奥林格斯技术有限公司 含有寡核苷酸的药物组合物及其用途
CA2898314A1 (en) 2001-07-19 2003-07-31 Perian Therapeutics, Inc. Multimeric proteins and methods of making and using same
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US20030096772A1 (en) 2001-07-30 2003-05-22 Crooke Rosanne M. Antisense modulation of acyl CoA cholesterol acyltransferase-2 expression
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
US20040143104A1 (en) * 2001-08-08 2004-07-22 Wadsworth Samuel C. Methods of treating diabetes and other blood sugar disorders
AU2002327430A1 (en) * 2001-08-08 2003-02-24 Genzyme Corporation Methods for treating diabetes and other blood sugar disorders
JP2005500060A (ja) * 2001-08-14 2005-01-06 レキシコン・ジェネティクス・インコーポレーテッド 新規ヒトコラーゲンタンパクおよびそれをコードするポリヌクレオチド
AU2002330015B2 (en) 2001-09-18 2008-02-07 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
US8735153B2 (en) 2001-09-24 2014-05-27 Sangamo Biosciences, Inc. Modulation of stem cells using zinc finger proteins
DE10244149A1 (de) * 2001-09-25 2003-04-30 Werner Keber Verfahren und Einrichtung zum Verhindern einer unzulässigen Flugannäherung von Flugzeugen an zu schützende Bodenobjeke
US7563879B2 (en) * 2001-09-26 2009-07-21 Intrexon Corporation Leafhopper ecdysone receptor nucleic acids, polypeptides, and uses thereof
MXPA04002808A (es) * 2001-09-26 2005-06-06 Rheogene Holdings Inc Acidos nucleicos receptores de ecdisona de mosca blanca, polipeptidos, y sus usos.
US20040023910A1 (en) * 2001-09-28 2004-02-05 Zhiming Zhang Use of cyr61 in the treatment and diagnosis of human uterine leiomyomas
US7906102B2 (en) 2001-10-03 2011-03-15 Vanderbilt University Ligands to radiation-induced molecules
CA2462876A1 (en) * 2001-10-05 2003-04-17 The Trustees Of The University Of Pennsylvania Compositions for and methods of treating and preventing sirs/sepsis
NZ566396A (en) 2001-10-09 2009-07-31 Isis Pharmaceuticals Inc Antisense modulation of insulin-like growth factor binding protein 5 expressions
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
MX339524B (es) 2001-10-11 2016-05-30 Wyeth Corp Composiciones inmunogenicas novedosas para la prevencion y tratamiento de enfermedad meningococica.
EP2325193A3 (en) 2001-11-02 2012-05-02 Insert Therapeutics, Inc. Methods and compositions for therapeutic use of RNA interference
AU2002348163A1 (en) * 2001-11-02 2003-05-19 Intradigm Corporation Therapeutic methods for nucleic acid delivery vehicles
US7488313B2 (en) * 2001-11-29 2009-02-10 Boston Scientific Scimed, Inc. Mechanical apparatus and method for dilating and delivering a therapeutic agent to a site of treatment
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
WO2003052095A2 (en) * 2001-12-14 2003-06-26 Transgene S.A. Use of a lysolipid for the preparation of a composition for transfection of a polynucleotide into a cell
PT1456377T (pt) * 2001-12-20 2019-09-10 Merck Sharp & Dohme Composições de syn3 e processos
US7255874B1 (en) 2001-12-21 2007-08-14 Closure Medical Corporation Biocompatible polymers and adhesives: compositions, methods of making and uses related thereto
EP2261250B1 (en) 2001-12-21 2015-07-01 Human Genome Sciences, Inc. GCSF-Albumin fusion proteins
CA2471431A1 (en) 2002-01-02 2003-07-17 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
CA2472927A1 (en) 2002-01-10 2003-07-17 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw A novel splice variant of myd88 and uses thereof
WO2003064625A2 (en) 2002-02-01 2003-08-07 Sequitur, Inc. Oligonucleotide compositions with enhanced efficiency
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
JP2005530695A (ja) * 2002-02-15 2005-10-13 ザイコス インク． 生理活性物質を細胞内に導入するエレクトロポレーション法
DE10207177A1 (de) * 2002-02-19 2003-09-04 Novosom Ag Fakultativ kationische Lipide
WO2003075889A1 (en) * 2002-03-05 2003-09-18 Transave, Inc. An inhalation system for prevention and treatment of intracellular infections
US9918665B2 (en) 2002-03-11 2018-03-20 Nitto Denko Corporation Transdermal porator and patch system and method for using same
WO2003101507A2 (en) 2002-03-11 2003-12-11 Altea Therapeutics Corporation Transdermal drug delivery device, method and use
US8116860B2 (en) 2002-03-11 2012-02-14 Altea Therapeutics Corporation Transdermal porator and patch system and method for using same
US20030180712A1 (en) 2002-03-20 2003-09-25 Biostratum Ab Inhibition of the beta3 subunit of L-type Ca2+ channels
AU2003230874A1 (en) 2002-04-16 2003-11-03 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
US20040180438A1 (en) * 2002-04-26 2004-09-16 Pachuk Catherine J. Methods and compositions for silencing genes without inducing toxicity
US20050240145A1 (en) * 2002-04-30 2005-10-27 Neal Scott Mechanical apparatus and method for dilating and delivering a therapeutic agent to a site of treatment
CA2495787A1 (en) * 2002-05-01 2003-11-13 Chromos Molecular Systems, Inc. Methods for delivering nucleic acid molecules into cells and assessment thereof
WO2003093449A2 (en) * 2002-05-06 2003-11-13 Nucleonics, Inc. Methods for delivery of nucleic acids
US7598421B2 (en) * 2002-05-08 2009-10-06 Ucl Biomedica Plc Materials for the delivery of biologically-active material to cells
US20040013649A1 (en) * 2002-05-10 2004-01-22 Inex Pharmaceuticals Corporation Cancer vaccines and methods of using the same
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
EA200401565A1 (ru) * 2002-05-24 2005-04-28 Неофарм, Инк. Способ получения кардиолипина или аналога кардиолипина (варианты), способ получения липосомы и композиция кардиолипина для лечения заболеваний (варианты)
EP2316922B1 (en) 2002-05-24 2013-05-22 Merck Sharp & Dohme Corp. Neutralizing human anti-IGFR antibody
CA2486967A1 (en) * 2002-05-24 2003-12-04 Neopharm, Inc. Cardiolipin compositions their methods of preparation and use
DK1519714T3 (da) 2002-06-28 2011-01-31 Protiva Biotherapeutics Inc Fremgangsmåde og apparat til fremstilling af liposomer
WO2004005464A2 (en) * 2002-07-02 2004-01-15 Uab Research Foundation Compounds promoting delivery of genes
WO2004004758A1 (en) * 2002-07-05 2004-01-15 Lipoxen Technologies Limited Method to enhance an immune response of nucleic acid vaccination
US7375093B2 (en) 2002-07-05 2008-05-20 Intrexon Corporation Ketone ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US20050239728A1 (en) * 2002-07-31 2005-10-27 Pachuk Catherine J Double stranded rna structures and constructs, and methods for generating and using the same
US20040034336A1 (en) * 2002-08-08 2004-02-19 Neal Scott Charged liposomes/micelles with encapsulted medical compounds
US7785608B2 (en) * 2002-08-30 2010-08-31 Wyeth Holdings Corporation Immunogenic compositions for the prevention and treatment of meningococcal disease
US7135324B2 (en) 2002-09-04 2006-11-14 The University Of Connecticut Viral recombinases, related articles, and methods of use thereof
WO2004022708A2 (en) * 2002-09-09 2004-03-18 The Brigham And Women's Hospital, Inc. Genes and proteins altering tau-related neurodegeneration
AU2003278776A1 (en) * 2002-09-10 2004-04-30 Vical Incorporated Codon-optimized polynucleotide-based vaccines against bacillus anthracis infection
EP2330194A3 (en) 2002-09-13 2011-10-12 Replicor, Inc. Non-sequence complementary antiviral oligonucleotides
US7148211B2 (en) * 2002-09-18 2006-12-12 Genzyme Corporation Formulation for lipophilic agents
US20040053895A1 (en) * 2002-09-18 2004-03-18 Bone Care International, Inc. Multi-use vessels for vitamin D formulations
AU2003302226A1 (en) * 2002-09-24 2004-06-30 University Of Kentucky Research Foundation Nanoparticle-based vaccine delivery system containing adjuvant
US7229976B2 (en) 2002-09-26 2007-06-12 Isis Pharmaceuticals, Inc. Modulation of forkhead box O1A expression
US9809654B2 (en) 2002-09-27 2017-11-07 Vaccinex, Inc. Targeted CD1d molecules
US20050277611A1 (en) * 2002-10-16 2005-12-15 Neopharm, Inc. Cationic cardiolipin analoges and its use thereof
US6863731B2 (en) * 2002-10-18 2005-03-08 Controls Corporation Of America System for deposition of inert barrier coating to increase corrosion resistance
KR101424980B1 (ko) * 2002-10-29 2014-08-01 인스메드 인코포레이티드 항감염제의 지속적인 방출
JP3447009B1 (ja) * 2002-10-29 2003-09-16 實 平垣 構築物用構成体およびその製造方法
US7879351B2 (en) * 2002-10-29 2011-02-01 Transave, Inc. High delivery rates for lipid based drug formulations, and methods of treatment thereof
US7718189B2 (en) 2002-10-29 2010-05-18 Transave, Inc. Sustained release of antiinfectives
TW200509968A (en) 2002-11-01 2005-03-16 Elan Pharm Inc Prevention and treatment of synucleinopathic disease
US8506959B2 (en) 2002-11-01 2013-08-13 Neotope Biosciences Limited Prevention and treatment of synucleinopathic and amyloidogenic disease
EP1560840B1 (en) 2002-11-05 2015-05-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
CA2504720C (en) 2002-11-05 2013-12-24 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
PT1569695E (pt) 2002-11-13 2013-07-16 Genzyme Corp Modulação anti-sentido da expressão de apolipoproteína
WO2004044181A2 (en) 2002-11-13 2004-05-27 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein b expression
US7144999B2 (en) 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
EP1903056A3 (en) 2002-12-10 2008-05-07 Idm Pharma, Inc. HLA-A1, -A2 -A3, -A24, -B7, and -B44 binding peptides comprising tumor associated antigen epitopes, and compositions thereof
WO2004060363A1 (en) * 2002-12-23 2004-07-22 Vical Incorporated Method for producing sterile polynucleotide based medicaments
EP1578193A4 (en) * 2002-12-23 2011-06-15 Vical Inc FREEZING PROCESS FOR NUCLEIC ACID / BLOCK COPOLYMER / CATION SIDE COMPLEXES
CA2756797C (en) 2002-12-23 2015-05-05 Vical Incorporated Codon-optimized polynucleotide-based vaccines against human cytomegalovirus infection
US20040138154A1 (en) * 2003-01-13 2004-07-15 Lei Yu Solid surface for biomolecule delivery and high-throughput assay
US20070269891A9 (en) * 2003-01-13 2007-11-22 Yasunobu Tanaka Solid surface with immobilized degradable cationic polymer for transfecting eukaryotic cells
EP1585815A4 (en) * 2003-01-21 2006-02-22 Bristol Myers Squibb Co A NEW ACYL-COENZYME A, MONOCLYLGLYCERIN ACYLTRANSFERASE-3 (MGAT3), CODING POLYNUCLEOTIDE, AND USES THEREOF
US7304161B2 (en) 2003-02-10 2007-12-04 Intrexon Corporation Diaclhydrazine ligands for modulating the expression of exogenous genes in mammalian systems via an ecdysone receptor complex
DK1597366T3 (da) 2003-02-11 2013-02-25 Antisense Therapeutics Ltd Modulering af ekspression af insulin-lignende vækstfaktor receptor I
WO2004074314A2 (en) * 2003-02-14 2004-09-02 University Of South Florida Chistosan-microparticles for ifn gene delivery
US7456315B2 (en) 2003-02-28 2008-11-25 Intrexon Corporation Bioavailable diacylhydrazine ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
EP2216407B1 (en) 2003-03-07 2016-01-13 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
WO2004092329A2 (en) * 2003-04-08 2004-10-28 Galenica Pharmaceuticals, Inc. Semi-synthetic saponin analogs with carrier and immune stimulatory activities for dna and rna vaccines
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US20070253964A1 (en) * 2003-04-16 2007-11-01 Zlotnick Gary W Novel Immunogenic Compositions for the Prevention and Treatment of Meningococcal Disease
CA2522184A1 (en) * 2003-04-17 2004-11-04 The Trustees Of Columbia University In The City Of New York Desmoglein 4 is a novel gene involved in hair growth
EP2666858A1 (en) 2003-04-17 2013-11-27 Alnylam Pharmaceuticals Inc. Modified iRNA agents
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
US8080642B2 (en) 2003-05-16 2011-12-20 Vical Incorporated Severe acute respiratory syndrome DNA compositions and methods of use
WO2004105697A2 (en) * 2003-05-22 2004-12-09 Molecular Transfer, Inc. Novel lipids for transfection of nucleic acids
BRPI0410886A (pt) 2003-06-03 2006-07-04 Isis Pharmaceuticals Inc composto de filamento duplo, composição farmacêutica, sal farmaceuticamente aceitável, métodos de modificação do ácido nucleico que codifica a survivina humana, de inibição da expressão da suvivina em células ou tecidos, e de tratamento de uma condição associada com a expressão ou superexpressão da suvivina, e, oligonucleotìdeo de rnai de filamento único
CN101396555A (zh) * 2003-06-04 2009-04-01 坎基股份有限公司 用于干扰素治疗的方法和组合物
CN1836042A (zh) 2003-06-12 2006-09-20 原子核物理公司 用于基因沉默的hbv和hcv保守序列
US7261882B2 (en) 2003-06-23 2007-08-28 Reagents Of The University Of Colorado Methods for treating neuropathic pain by administering IL-10 polypeptides
US20060078560A1 (en) * 2003-06-23 2006-04-13 Neopharm, Inc. Method of inducing apoptosis and inhibiting cardiolipin synthesis
EP2567693B1 (en) * 2003-07-16 2015-10-21 Protiva Biotherapeutics Inc. Lipid encapsulated interfering RNA
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2534296C (en) 2003-08-08 2013-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7330851B2 (en) * 2003-08-18 2008-02-12 Eaglehawk, Limited Data security through dissembly of data elements or connections between elements
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
JP2007503205A (ja) * 2003-08-22 2007-02-22 ニュークレオニクス・インコーポレイテッド 多コンパートメント真核生物発現系
US7291604B2 (en) * 2003-09-03 2007-11-06 The General Hospital Corporation Methods of treating restenosis
US7803397B2 (en) * 2003-09-15 2010-09-28 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
US7425544B2 (en) 2003-09-18 2008-09-16 Eli Lilly And Company Modulation of eIF4E expression
US7407776B2 (en) 2003-09-19 2008-08-05 Sangamo Biosciences, Inc. Engineered zinc finger proteins for regulation of gene expression
US7662929B2 (en) 2003-10-10 2010-02-16 Alchemia Oncology Pty Limited Antibody that specifically binds hyaluronan synthase
DE10347710B4 (de) 2003-10-14 2006-03-30 Johannes-Gutenberg-Universität Mainz Rekombinante Impfstoffe und deren Verwendung
WO2005048988A1 (en) * 2003-10-22 2005-06-02 Neal Scott Charged liposomes/micelles with encapsulated medical compounds
US8016811B2 (en) 2003-10-24 2011-09-13 Altea Therapeutics Corporation Method for transdermal delivery of permeant substances
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
ITMI20032185A1 (it) * 2003-11-12 2005-05-13 Chemi Spa Processo per la sintesi di lipidi cationici.
US20050107318A1 (en) * 2003-11-17 2005-05-19 Samuel Wadsworth Methods of treating diabetes and other blood sugar disorders
SI2295073T1 (sl) 2003-11-17 2014-07-31 Genentech, Inc. Protitelo proti CD22 za zdravljenje tumorja hematopoetskega izvora
WO2005061717A1 (ja) * 2003-12-19 2005-07-07 Dainippon Sumitomo Pharma Co., Ltd. 新規な核酸導入法
SE0303588D0 (sv) 2003-12-30 2003-12-30 Bioactive Polymers Ab C O Lund Surface protection of exposed biological tissues
WO2005071080A2 (en) 2004-01-20 2005-08-04 Isis Pharmaceuticals, Inc. Modulation of glucocorticoid receptor expression
US7468431B2 (en) 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US7432057B2 (en) * 2004-01-30 2008-10-07 Michigan State University Genetic test for PSE-susceptible turkeys
WO2005080594A2 (en) * 2004-02-24 2005-09-01 Innogenetics N.V. Method for determining the risk of developing a neurological disease
US8569474B2 (en) 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
WO2005089268A2 (en) 2004-03-15 2005-09-29 Isis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of rna by rnase h
WO2005098049A2 (en) 2004-03-25 2005-10-20 California Institute Of Technology Hybridization chain reaction
ES2707393T3 (es) * 2004-03-26 2019-04-03 Curis Inc Moduladores de interferencia de ARN de señalización de hedgehog y usos de los mismos
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
JP4564052B2 (ja) 2004-04-08 2010-10-20 サンガモ バイオサイエンシズ インコーポレイテッド 心筋収縮能調節用の方法及び組成物
US20050260652A1 (en) * 2004-04-15 2005-11-24 The General Hospital Corporation Compositions and methods that modulate RNA interference
EP1745295B1 (en) 2004-04-20 2010-10-06 Galapagos N.V. Methods, compositions and compound assays for inhibiting amyloid-beta protein production
EP1747462A2 (en) 2004-04-27 2007-01-31 Galapagos N.V. Methods, agents, and compound screening assays for inducing differentiation of undifferentiated mammalian cells into osteoblasts
US7935510B2 (en) * 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
US7303881B2 (en) * 2004-04-30 2007-12-04 Pds Biotechnology Corporation Antigen delivery compositions and methods of use
KR101462819B1 (ko) 2004-05-03 2014-11-21 헤르메스 바이오사이언스, 인코포레이티드 약물 전달에 유용한 리포좀
JP5331340B2 (ja) 2004-05-18 2013-10-30 バイカル インコーポレイテッド インフルエンザウィルスワクチン組成物、及びその使用方法
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
ATE537263T1 (de) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Kationische lipide und verwendungsverfahren
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
EP2256198A1 (en) 2004-06-14 2010-12-01 Galapagos N.V. Methods for identification, and compounds useful for the treatment of degenerative and inflammatory diseases
EP1759203A2 (en) 2004-06-21 2007-03-07 Galapagos N.V. Methods and means for treatment of osteoarthritis
US7094775B2 (en) 2004-06-30 2006-08-22 Bone Care International, Llc Method of treating breast cancer using a combination of vitamin D analogues and other agents
CA2572439A1 (en) * 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory sirna molecules and uses therefor
US20080299095A1 (en) * 2004-07-09 2008-12-04 Bc Cancer Agency Nup98-Hox Fusions for Expansion of Hemopoietic Stem Cells
US7604798B2 (en) * 2004-07-15 2009-10-20 Northwestern University Methods and compositions for importing nucleic acids into cell nuclei
AU2005274948B2 (en) 2004-07-16 2011-09-22 Genvec, Inc. Vaccines against aids comprising CMV/R-nucleic acid constructs
US20060051405A1 (en) * 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
EA013752B1 (ru) 2004-08-09 2010-06-30 Элан Фармасьютикалз, Инк. Предупреждение и лечение синуклеинопатических и амилоидогенных заболеваний
ATE483815T1 (de) 2004-08-23 2010-10-15 Alnylam Pharmaceuticals Inc Expressionskonstrukte mit mehreren rna-polymerase-iii-promotoren
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
US20060063231A1 (en) 2004-09-16 2006-03-23 Sangamo Biosciences, Inc. Compositions and methods for protein production
EP2325314B1 (en) 2004-09-24 2014-08-20 Alnylam Pharmaceuticals, Inc. Targeting opposite strand replication intermediates of single-stranded viruses by RNAI
EP1799825B1 (en) 2004-10-05 2011-06-29 The California Institute of Technology Aptamer regulated nucleic acids and uses thereof
US7485468B2 (en) 2004-10-15 2009-02-03 Galapagos Bv Molecular targets and compounds, and methods to identify the same, useful in the treatment of joint degenerative and inflammatory diseases
US20090281025A1 (en) * 2004-10-18 2009-11-12 Mount Sinai School Of Medicine Of New York University Inhibition of tumor growth and metastasis by atf2-derived peptides
AU2005302554A1 (en) * 2004-10-28 2006-05-11 Idexx Laboratories, Inc. Compositions for controlled delivery of pharmaceutically active compounds
US20060147449A1 (en) * 2004-11-15 2006-07-06 Brass Lawrence F Method of using CD100 (or Sema4D) to mediate platelet activation and inflammatory responses
EP2199298A1 (en) * 2004-11-17 2010-06-23 Protiva Biotherapeutics Inc. Sirna silencing of Apolipoprotein B
DE102004057303A1 (de) * 2004-11-26 2006-06-01 Merck Patent Gmbh Stabile Kristallmodifikationen von DOTAP Chlorid
WO2006060723A2 (en) * 2004-12-03 2006-06-08 Vical Incorporated Methods for producing block copolymer/amphiphilic particles
AU2005319306B9 (en) 2004-12-22 2012-04-05 Alnylam Pharmaceuticals, Inc. Conserved HBV and HCV sequences useful for gene silencing
US9034650B2 (en) 2005-02-02 2015-05-19 Intrexon Corporation Site-specific serine recombinases and methods of their use
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
EP2314614B1 (en) 2005-02-28 2015-11-25 Sangamo BioSciences, Inc. Anti-angiogenic methods and compositions
US7727721B2 (en) * 2005-03-08 2010-06-01 California Institute Of Technology Hybridization chain reaction amplification for in situ imaging
JP2008535796A (ja) 2005-03-10 2008-09-04 ジェネンテック・インコーポレーテッド 脈管の完全性を調節するための方法及び組成物
US20090062184A1 (en) * 2005-03-24 2009-03-05 Dainippon Sumitomo Pharma Co., Ltd. Fine particulate preparation comprising complex of nucleic acid molecule and collagen
CA2603730A1 (en) 2005-03-31 2006-10-05 Calando Pharmaceuticals, Inc. Inhibitors of ribonucleotide reductase subunit 2 and uses thereof
US20070026061A1 (en) * 2005-05-25 2007-02-01 Nahid Ali Liposomal formulation and use thereof
EP2816118B1 (en) 2005-05-31 2018-10-17 The Regents of the University of Colorado, a body corporate Methods for delivering genes
JP5140579B2 (ja) 2005-06-08 2013-02-06 カンジェーン コーポレイション 病原菌に対する生体防御を増強するヒアルロン酸結合性ペプチド
WO2007005898A2 (en) * 2005-07-05 2007-01-11 Cornell Research Foundation, Inc. Blocking leukocyte emigration and inflammation by interfering with cd99l2
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
US20070054873A1 (en) * 2005-08-26 2007-03-08 Protiva Biotherapeutics, Inc. Glucocorticoid modulation of nucleic acid-mediated immune stimulation
US7666584B2 (en) * 2005-09-01 2010-02-23 Philadelphia Health & Education Coporation Identification of a pin specific gene and protein (PIN-1) useful as a diagnostic treatment for prostate cancer
EP1931789B1 (en) 2005-09-20 2016-05-04 BASF Plant Science GmbH Methods for controlling gene expression using ta-siran
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
US7312079B1 (en) 2005-10-06 2007-12-25 Lexicon Pharmaceuticals, Inc. Variants of FAM3C
US7960357B2 (en) * 2005-10-07 2011-06-14 California Institute Of Technology PKR activation via hybridization chain reaction
JP5671206B2 (ja) 2005-10-21 2015-02-18 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 黒色腫におけるｃ−ＫＩＴ発癌遺伝子の変異
US8101741B2 (en) * 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US7754679B2 (en) * 2005-11-16 2010-07-13 Idexx Laboratories, Inc. Pharmaceutical compositions for the administration of aptamers
US8114440B2 (en) 2005-11-16 2012-02-14 Idexx Laboratories Inc. Pharmaceutical compositions for the administration of aptamers
US7807652B2 (en) 2005-11-21 2010-10-05 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
TWI461436B (zh) 2005-11-25 2014-11-21 Kyowa Hakko Kirin Co Ltd 人類ｃｄ１３４（ｏｘ４０）之人類單株抗體及其製造及使用方法
ES2594368T3 (es) 2005-12-08 2016-12-19 Insmed Incorporated Composiciones a base de lípidos de antiinfecciosos para tratar infecciones pulmonares
US10647960B2 (en) * 2005-12-13 2020-05-12 The Trustees Of The University Of Pennsylvania Transcriptome transfer produces cellular phenotype conversion
WO2007070705A2 (en) 2005-12-15 2007-06-21 The Trustees Of The University Of Pennsylvania Cationic lipid-mediated vectors
CA2632331C (en) 2005-12-22 2015-02-10 Vib Vzw Molecules with a beta-aggregating region and their use in inducing protein aggregation
WO2007073489A2 (en) * 2005-12-22 2007-06-28 Trustees Of Boston University Molecules for gene delivery and gene therapy, and methods of use thereof
US8669418B2 (en) 2005-12-22 2014-03-11 Vib Vzw Means and methods for mediating protein interference
PL2161038T3 (pl) 2006-01-26 2014-06-30 Ionis Pharmaceuticals Inc Kompozycje i ich zastosowanie ukierunkowane na huntingtynę
EP1981838B1 (en) * 2006-02-07 2015-12-02 Council of Scientific and Industrial Research Process for synthesis of glycomimicking cationic amphiphiles
JP2009525757A (ja) 2006-02-10 2009-07-16 ザ ユニバーシティ オブ シンシナティ 心機能のレギュレータとしてのホスファターゼインヒビタープロテイン−１
CA2647277A1 (en) 2006-04-05 2007-11-08 Genentech, Inc. Method for using boc/cdo to modulate hedgehog signaling
EP2015785A4 (en) * 2006-04-24 2009-07-15 Shantha West Inc ANTIGEN AGRM2
JP2009535383A (ja) 2006-05-03 2009-10-01 バルティック テクロノジー デヴェロプメント，リミテッド 強く結合した塩基で修飾されたオリゴヌクレオチドと人工ヌクレアーゼを組み合わせたアンチセンス作用物質
EP2604255B1 (en) 2006-05-05 2017-10-25 Molecular Transfer, Inc. Novel reagents for transfection of eukaryotic cells
EP2213731B1 (en) 2006-05-25 2013-12-04 Sangamo BioSciences, Inc. Variant foki cleavage half-domains
GB0610636D0 (en) 2006-05-30 2006-07-05 Univ London Materials and complexes for the delivery of biologically-active material to cells
CA2653628C (en) 2006-06-01 2015-07-14 Elan Pharmaceuticals, Inc. Neuroactive fragments of app
US7915399B2 (en) * 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US7572618B2 (en) 2006-06-30 2009-08-11 Bristol-Myers Squibb Company Polynucleotides encoding novel PCSK9 variants
US20080004410A1 (en) * 2006-06-30 2008-01-03 Yu-Chin Lai Hydrophilic macromonomers having alpha,beta-conjugated carboxylic terminal group and medical devices incorporating same
JP5072275B2 (ja) 2006-07-03 2012-11-14 テルモ株式会社 閉鎖小胞の分離方法、製剤の製造方法および評価方法
WO2008011473A2 (en) 2006-07-19 2008-01-24 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hbxip
JP2009544731A (ja) * 2006-07-28 2009-12-17 サノフィ−アベンティス 腫瘍治療のための組成物及び方法
CN101588816B (zh) 2006-10-19 2013-06-19 Csl有限公司 白介素-13受体α1的高亲和性抗体拮抗物
ES2388567T3 (es) 2006-10-19 2012-10-16 Csl Limited Anticuerpos anti-il-13r alfa 1 y usos de los mismos
WO2008070350A2 (en) * 2006-10-27 2008-06-12 The Board Of Regents Of The University Of Texas System Methods and compositions related to wrapping of dehydrons
WO2008058291A2 (en) 2006-11-09 2008-05-15 California Institute Of Technology Modular aptamer-regulated ribozymes
EP2101813B1 (en) 2006-11-27 2014-04-02 Patrys Limited Novel glycosylated peptide target in neoplastic cells
EP1938843A1 (en) * 2006-12-19 2008-07-02 Novosom AG Lipids and lipid assemblies comrising transfection enhancer elements
AR064642A1 (es) * 2006-12-22 2009-04-15 Wyeth Corp Polinucleotido vector que lo comprende celula recombinante que comprende el vector polipeptido , anticuerpo , composicion que comprende el polinucleotido , vector , celula recombinante polipeptido o anticuerpo , uso de la composicion y metodo para preparar la composicion misma y preparar una composi
US8048998B2 (en) * 2007-01-19 2011-11-01 Exiqon A/S Mediated cellular delivery of LNA oligonucleotides
EP2641971A1 (en) 2007-01-29 2013-09-25 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2009045469A2 (en) 2007-10-02 2009-04-09 Amgen Inc. Increasing erythropoietin using nucleic acids hybridizable to micro-rna and precursors thereof
US9603922B2 (en) 2007-02-21 2017-03-28 Vaccinex, Inc. Modulation of NKT cell activity with antigen-loaded CD1d molecules
AU2008218199B2 (en) 2007-02-22 2013-10-31 Genentech, Inc. Methods for detecting inflammatory bowel disease
ES2546863T3 (es) 2007-02-23 2015-09-29 Prothena Biosciences Limited Prevención y tratamiento de enfermedad sinucleinopática y amiloidogénica
EP2118300B1 (en) 2007-02-23 2015-05-27 Prothena Biosciences Limited Prevention and treatment of synucleinopathic and amyloidogenic disease
US8318921B2 (en) 2007-03-01 2012-11-27 California Institute Of Technology Triggered RNAi
WO2008106646A2 (en) * 2007-03-01 2008-09-04 Introgen Therapeutics, Inc Methods and formulations for topical gene therapy
US8877206B2 (en) * 2007-03-22 2014-11-04 Pds Biotechnology Corporation Stimulation of an immune response by cationic lipids
DE102007015598A1 (de) * 2007-03-29 2008-10-02 Heinrich-Heine-Universität Düsseldorf Verwendung von fluorhaltigen Verbindungen zu Diagnosezwecken mit Hilfe bildgebender Verfahren
WO2008124768A1 (en) 2007-04-09 2008-10-16 The General Hospital Corporation Hemojuvelin fusion proteins and uses thereof
CN101674853B (zh) 2007-05-04 2013-03-27 玛瑞纳生物技术有限公司 氨基酸脂质及其用途
US20100196455A1 (en) 2007-05-04 2010-08-05 Transave, Inc. Compositions of Multicationic Drugs for Reducing Interactions with Polyanionic Biomolecules and Methods of Use Thereof
US9114081B2 (en) 2007-05-07 2015-08-25 Insmed Incorporated Methods of treating pulmonary disorders with liposomal amikacin formulations
US9333214B2 (en) 2007-05-07 2016-05-10 Insmed Incorporated Method for treating pulmonary disorders with liposomal amikacin formulations
US9119783B2 (en) 2007-05-07 2015-09-01 Insmed Incorporated Method of treating pulmonary disorders with liposomal amikacin formulations
US9217151B2 (en) 2007-05-16 2015-12-22 California Institute Of Technology Versatile nucleic acid hairpin motif for programming biomolecular self-assembly pathways
CA2687115A1 (en) * 2007-05-16 2008-11-20 Mat Malta Advanced Technologies Limited Treatment and prevention of influenza
CA2688346A1 (en) * 2007-05-23 2008-12-04 Vical Incorporated Compositions and methods for enhancing immune responses to vaccines
MX2009012834A (es) 2007-05-29 2010-05-17 Intrexon Corp Ligandos quirales de diacilhidrazina para modular la expresion de genes exogenos por medio de un complejo receptor de ecdisona.
US20080312174A1 (en) * 2007-06-05 2008-12-18 Nitto Denko Corporation Water soluble crosslinked polymers
EP2162747B1 (en) 2007-06-20 2014-04-30 Galapagos N.V. Molecular targets and methods to identify compounds for treating bone and joint degenerative diseases
US20090069266A1 (en) * 2007-06-27 2009-03-12 Northwestern University Methods and compositions for nucleic acid transfer into cells
WO2009009086A2 (en) 2007-07-12 2009-01-15 Sangamo Biosciences, Inc. Methods and compositions for inactivating alpha 1,6 fucosyltransferase (fut 8) gene expression
WO2009011855A2 (en) * 2007-07-16 2009-01-22 California Institute Of Technology Selection of nucleic acid-based sensor domains within nucleic acid switch platform
US8828960B2 (en) * 2007-07-17 2014-09-09 Idexx Laboratories, Inc. Amino acid vitamin ester compositions for controlled delivery of pharmaceutically active compounds
AU2008289461A1 (en) 2007-08-23 2009-02-26 Intrexon Corporation Methods and compositions for diagnosing disease
US8367815B2 (en) 2007-08-28 2013-02-05 California Institute Of Technology Modular polynucleotides for ligand-controlled regulatory systems
US20120165387A1 (en) 2007-08-28 2012-06-28 Smolke Christina D General composition framework for ligand-controlled RNA regulatory systems
US8865667B2 (en) 2007-09-12 2014-10-21 California Institute Of Technology Higher-order cellular information processing devices
AU2008302504B2 (en) * 2007-09-17 2014-08-14 Agrofresh Inc. Compositions and methods for the modification of physiological responses in plants
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
JP2010540534A (ja) 2007-09-28 2010-12-24 イントレキソン コーポレーション 生体治療分子の発現のための治療遺伝子スイッチ構築物およびバイオリアクター、ならびにその使用
CA2971794C (en) 2007-10-04 2020-03-24 Zymogenetics, Inc. B7 family member zb7h6 and related compositions and methods
AU2008311292B9 (en) 2007-10-08 2014-10-09 Intrexon Corporation Engineered dendritic cells and uses for the treatment of cancer
GB0720486D0 (en) * 2007-10-19 2007-11-28 Univ Edinburgh Cationic lipids
EP2217721A4 (en) * 2007-10-29 2013-01-09 Univ California GENE THERAPY FOR OSTEOARTHRITIS
US8097712B2 (en) 2007-11-07 2012-01-17 Beelogics Inc. Compositions for conferring tolerance to viral disease in social insects, and the use thereof
DE102007056488A1 (de) 2007-11-22 2009-07-23 Biontex Laboratories Gmbh Steigerung von Transfektionseffizienzen nicht-viraler Genliefersysteme durch Blockierung des angeborenen Immunsystems
CN101918566A (zh) 2007-11-22 2010-12-15 Biontex实验室有限公司 通过影响先天性免疫系统改进非病毒输送系统的转染结果
DE102008023913A1 (de) 2008-05-16 2009-11-19 Biontex Laboratories Gmbh Verbesserung von Transfektionsergebnissen nicht-viraler Genliefersysteme durch Beeinflussung des angeborenen Immunsystems
DE102008016275A1 (de) 2008-03-28 2009-11-19 Biontex Laboratories Gmbh Verbesserung von Transfektionsergebnissen nicht-viraler Genliefersysteme durch Blockierung des angeborenen Immunsystems
CA3043911A1 (en) 2007-12-04 2009-07-02 Arbutus Biopharma Corporation Targeting lipids
US9029524B2 (en) 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
CA2904904A1 (en) 2007-12-11 2009-06-18 The Scripps Research Institute Compositions and methods related to mrna translational enhancer elements
WO2009078799A1 (en) 2007-12-17 2009-06-25 Marfl Ab New vaccine for the treatment of mycobacterium related disorders
ES2535419T3 (es) 2007-12-27 2015-05-11 Protiva Biotherapeutics Inc. Silenciamiento de expresión de quinasa tipo polo usando ARN interferente
EA036059B1 (ru) 2007-12-28 2020-09-21 Протена Байосайенсиз Лимитед Способ получения антитела или его фрагмента, которое специфически связывается с агрегированным амилоидным белком
CN102016036B (zh) 2008-02-11 2015-04-08 阿克赛医药公司 经修饰的RNAi多核苷酸及其用途
US8497364B2 (en) * 2008-02-27 2013-07-30 California Institute Of Technology Triggered RNAi
US20110045001A1 (en) * 2008-03-28 2011-02-24 Biontex Laboratories Gmbh Transfection results of non-viral gene delivery systems by influencing of the innate immune system
BRPI0911332A2 (pt) 2008-04-04 2019-09-24 Calando Pharmaceuticals Inc composições e uso de inibidores de epas1
JP5788312B2 (ja) 2008-04-11 2015-09-30 アルニラム ファーマスーティカルズ インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達
WO2009129319A2 (en) 2008-04-15 2009-10-22 Protiva Biotherapeutics, Inc. Silencing of csn5 gene expression using interfering rna
HUE034483T2 (en) 2008-04-15 2018-02-28 Protiva Biotherapeutics Inc New lipid preparations for introducing a nucleic acid
JP5971945B2 (ja) 2008-04-17 2016-08-17 ピーディーエス バイオテクノロジー コーポレイションＰｄｓ Ｂｉｏｔｅｃｈｎｏｌｏｇｙ Ｃｏｒｐｏｒａｔｉｏｎ カチオン性脂質の鏡像異性体による免疫応答の刺激
US20100021904A1 (en) * 2008-05-21 2010-01-28 Pierce Niles A Shielded cross-linking probes
US20100021901A1 (en) * 2008-05-22 2010-01-28 Peng Yin Compositions and methods for detecting analytes
US8241854B2 (en) * 2008-05-22 2012-08-14 California Institute Of Technology Triggered RNAi
WO2009155100A1 (en) 2008-05-30 2009-12-23 Yale University Targeted oligonucleotide compositions for modifying gene expression
CA2893175C (en) 2008-06-10 2016-09-06 Sangamo Biosciences, Inc. Methods and compositions for generation of bax- and bak-deficient cell lines
MX2010014358A (es) 2008-06-20 2011-07-04 Wyeth Llc Composiciones y metodos de uso del orf1358 de cepas estreptococicas beta-hemoliticas.
WO2010008582A2 (en) 2008-07-18 2010-01-21 Rxi Pharmaceuticals Corporation Phagocytic cell drug delivery system
CA2734235C (en) 2008-08-22 2019-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
US8252762B2 (en) 2008-08-25 2012-08-28 Excaliard Pharmaceuticals, Inc. Antisense oligonucleotides directed against connective tissue growth factor and uses thereof
EP2352828B1 (en) 2008-09-11 2015-02-25 Galapagos N.V. Method for identifying compounds useful for increasing the functional activity and cell surface expression of cf-associated mutant cystic fibrosis transmembrane conductance regulator
EP3336188B1 (en) 2008-09-22 2020-05-06 Phio Pharmaceuticals Corp. Reduced size self-delivering rnai compounds
EP2350043B9 (en) 2008-10-09 2014-08-20 TEKMIRA Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
US20100112042A1 (en) 2008-10-16 2010-05-06 Mdrna, Inc. Processes and Compositions for Liposomal and Efficient Delivery of Gene Silencing Therapeutics
JP2012508174A (ja) 2008-11-05 2012-04-05 ワイス・エルエルシー β溶血性連鎖球菌（ＢＨＳ）疾患を予防するための多成分免疫原性組成物
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
CN102361985B (zh) 2008-12-04 2017-06-20 库尔纳公司 通过抑制肿瘤抑制基因的天然反义转录物治疗肿瘤抑制基因相关性疾病
JP6099868B2 (ja) 2008-12-04 2017-03-22 クルナ・インコーポレーテッド サーチュイン１（ｓｉｒｔ１）に対する天然アンチセンス転写物の抑制によるサーチュイン１関連疾患の治療
MX2011005910A (es) 2008-12-04 2011-06-17 Opko Curna Llc Tratamiento de enfermedades relacionadas con eritropoyetina (epo) mediante inhibicion del transcrito antisentido natural a eritropoyetina.
EP3156494B8 (en) 2008-12-04 2018-09-19 Sangamo Therapeutics, Inc. Genome editing in rats using zinc-finger nucleases
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
FR2941152B1 (fr) * 2009-01-20 2013-10-18 Centre Nat Rech Scient Vecteurs comprenant une macromolecule anionique et un lipide cationique pour l'administration de petits acides nucleiques.
EP2393506B1 (en) 2009-02-04 2018-03-28 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathies
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
EP2396408B1 (en) 2009-02-12 2017-09-20 CuRNA, Inc. Treatment of glial cell derived neurotrophic factor (gdnf) related diseases by inhibition of natural antisense transcript to gdnf
PT2396038E (pt) 2009-02-12 2016-02-19 Curna Inc Tratamento das doenças associadas com o factor neurotrófico derivado do cérebro (bdnf) por inibição do produto antisenso natural da transcrição para bdnf
WO2010096561A1 (en) 2009-02-18 2010-08-26 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic hiv/siv gag proteins and uses thereof
US8329882B2 (en) 2009-02-18 2012-12-11 California Institute Of Technology Genetic control of mammalian cells with synthetic RNA regulatory systems
EP2398479A2 (en) 2009-02-19 2011-12-28 Galapagos N.V. Methods for identifying and compounds useful for the diagnosis and treatment of diseases involving inflammation
JP2012517821A (ja) 2009-02-19 2012-08-09 ガラパゴス・ナムローゼ・フェンノートシャップ 炎症を包含する疾患の診断及び治療に有用な同定方法及び化合物
WO2010094732A1 (en) 2009-02-19 2010-08-26 Biofocus Dpi B.V. Methods for identifying and compounds useful for the diagnosis and treatment of diseases involving inflammation
WO2010102058A2 (en) 2009-03-04 2010-09-10 Curna, Inc. Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirt 1
JP6116242B2 (ja) 2009-03-16 2017-04-19 クルナ・インコーポレーテッド 核因子（赤血球由来２）様２（ｎｒｆ２）に対する天然アンチセンス転写物の抑制によるｎｒｆ２関連疾患の治療
US9708604B2 (en) 2009-03-17 2017-07-18 Curna, Inc. Treatment of delta-like 1 homolog (DLK1) related diseases by inhibition of natural antisense transcript to DLK1
WO2010115825A2 (en) 2009-03-31 2010-10-14 Robert Zimmermann Modulation of adipose triglyceride lipase for prevention and treatment of cachexia, loss of weight and muscle atrophy and methods of screening therefor
WO2010112569A1 (en) 2009-03-31 2010-10-07 Robert Zimmermann Modulation of adipose triglyceride lipase for prevention and treatment of cachexia, loss of weight and muscle atrophy and methods of screening therefor
JP2012522977A (ja) 2009-04-01 2012-09-27 ガラパゴス・ナムローゼ・フェンノートシャップ 変形性関節症の治療のための方法及び手段
US9145555B2 (en) 2009-04-02 2015-09-29 California Institute Of Technology Integrated—ligand-responsive microRNAs
JP6215533B2 (ja) 2009-04-09 2017-10-18 サンガモ セラピューティクス， インコーポレイテッド 幹細胞への標的組込み
EP2417984B1 (en) 2009-04-10 2016-03-30 Kyowa Hakko Kirin Co., Ltd. Method for treatment of blood tumor using anti-tim-3 antibody
CN102724993B (zh) 2009-04-17 2016-04-27 纽约大学 靶向tnf家族受体并拮抗tnf作用的肽、组合物、方法及其用途
EP3248618A1 (en) 2009-04-22 2017-11-29 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
US20100273203A1 (en) * 2009-04-23 2010-10-28 Board Of Trustees Of The University Of Arkansas Methods and compositions for detecting metabolites
CN104558179A (zh) 2009-04-27 2015-04-29 协和发酵麒麟株式会社 用于治疗血液肿瘤的抗IL-3Rα抗体
US8044215B2 (en) * 2009-04-29 2011-10-25 Juvaris Biotherapeutics, Inc. Methods for the preparation of amphillic nitrogen containing imidazolinium derivative compounds
EP2424987B1 (en) 2009-05-01 2017-11-15 CuRNA, Inc. Treatment of hemoglobin (hbf/hbg) related diseases by inhibition of natural antisense transcript to hbf/hbg
KR101835889B1 (ko) 2009-05-06 2018-03-08 큐알엔에이, 인크. 지질 수송 및 대사 유전자에 대한 천연 안티센스 전사체의 억제에 의해 지질 수송 및 대사 유전자 관련된 질환의 치료
JP6250930B2 (ja) 2009-05-06 2017-12-20 クルナ・インコーポレーテッド トリステトラプロリン（ｔｔｐ）に対する天然アンチセンス転写物の抑制によるｔｔｐ関連疾患の治療
WO2010135207A1 (en) 2009-05-16 2010-11-25 Agave Pharma, Incorporated Compositions comprising cationic amphiphiles and colipids for delivering therapeutics molecules
NO2432881T3 (es) 2009-05-18 2018-04-14
CN102549158B (zh) 2009-05-22 2017-09-26 库尔纳公司 通过抑制针对转录因子e3(tfe3)的天然反义转录物来治疗tfe3和胰岛素受体底物蛋白2(irs2)相关的疾病
US8791085B2 (en) 2009-05-28 2014-07-29 Curna, Inc. Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
CA2797480A1 (en) 2009-06-15 2010-12-23 4S3 Bioscience Inc. Methods and compositions for treatment of myotubular myopathy using chimeric polypeptides comprising myotubularin 1 (mtm1) polypeptides
CN102695797B (zh) 2009-06-16 2018-05-25 库尔纳公司 通过抑制针对胶原基因的天然反义转录物来治疗胶原基因相关的疾病
CN102612560B (zh) 2009-06-16 2017-10-17 库尔纳公司 通过抑制针对对氧磷酶1(pon1)的天然反义转录物来治疗pon1相关的疾病
ES2618894T3 (es) 2009-06-24 2017-06-22 Curna, Inc. Tratamiento de enfermedades relacionadas con el receptor del factor de necrosis tumoral 2 (tnfr2) por inhibición del transcrito natural antisentido para tnfr2
JP5907866B2 (ja) 2009-06-26 2016-04-26 クルナ・インコーポレーテッド ダウン症候群遺伝子に対する天然アンチセンス転写物の抑制によるダウン症候群遺伝子関連疾患の治療
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
EP2823810B9 (en) 2009-07-09 2019-10-30 Novosom Verwaltungs GmbH Amphoteric liposomes comprising imino lipids
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
US9234016B2 (en) 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
DK2462230T3 (en) 2009-08-03 2015-10-19 Recombinetics Inc METHODS AND COMPOSITIONS FOR TARGETED RE-MODIFICATION
WO2011015572A1 (en) 2009-08-03 2011-02-10 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
WO2011015573A1 (en) 2009-08-03 2011-02-10 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
ES2585360T3 (es) 2009-08-05 2016-10-05 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen de la insulina (INS) por inhibición de la transcripción antisentido natural en un gen de la insulina (INS)
US8410064B2 (en) * 2009-08-24 2013-04-02 The Board Of Trustees Of The University Of Arkansas Classical cannabinoid metabolites and methods of use thereof
CN102482671B (zh) 2009-08-25 2017-12-01 库尔纳公司 通过抑制‘含有iq模体的gtp酶激活蛋白’(iqgap)的天然反义转录物而治疗iqgap相关疾病
US9321823B2 (en) 2009-09-02 2016-04-26 Genentech, Inc. Mutant smoothened and methods of using the same
US9222086B2 (en) 2009-09-23 2015-12-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
AU2010306940A1 (en) 2009-10-12 2012-06-07 Smith, Larry Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
US8962584B2 (en) 2009-10-14 2015-02-24 Yissum Research Development Company Of The Hebrew University Of Jerusalem, Ltd. Compositions for controlling Varroa mites in bees
ES2564207T3 (es) 2009-10-22 2016-03-18 F. Hoffmann-La Roche Ag Métodos y composiciones para modular la activación con hepsina de la proteína estimuladora de macrófagos
WO2011056682A1 (en) 2009-10-27 2011-05-12 The University Of British Columbia Reverse head group lipids, lipid particle compositions comprising reverse headgroup lipids, and methods for the delivery of nucleic acids
WO2011056234A1 (en) 2009-11-06 2011-05-12 Fibrogen, Inc. Treatment for radiation-induced disorders
WO2011060233A1 (en) 2009-11-11 2011-05-19 The Trustees Of The University Of Pennsylvania Anti-tem1 antibodies and uses thereof
EP3002297B1 (en) 2009-11-30 2020-04-08 F. Hoffmann-La Roche AG Antibodies for treating and diagnosing tumors expressing slc34a2 (tat211)
PL3338765T3 (pl) 2009-12-01 2019-06-28 Translate Bio, Inc. Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych
CN104529945A (zh) 2009-12-11 2015-04-22 基因密码公司 使用gdnf家族配体(gfl)模拟剂或ret信号传导通路活化剂促进神经细胞存活的方法
ES2661813T3 (es) 2009-12-16 2018-04-04 Curna, Inc. Tratamiento de enfermedades relacionadas con la peptidasa del factor de transcripción de membrana, sitio 1 (mbtps1) mediante inhibición del transcrito antisentido natural al gen mbtps1
KR101793753B1 (ko) 2009-12-23 2017-11-03 큐알엔에이, 인크. 커플링방지 단백질 ２(ｕｃｐ２)에 대한 천연 안티센스 전사체의 저해에 의한 ｕｃｐ２ 관련 질환의 치료
KR101891352B1 (ko) 2009-12-23 2018-08-24 큐알엔에이, 인크. 간세포 성장 인자(ｈｇｆ)에 대한 천연 안티센스 전사체의 억제에 의한 ｈｇｆ 관련 질환의 치료
RU2611186C2 (ru) 2009-12-29 2017-02-21 Курна, Инк. ЛЕЧЕНИЕ ЗАБОЛЕВАНИЙ, СВЯЗАННЫХ С ОПУХОЛЕВЫМ БЕЛКОМ 63 (р63), ПУТЕМ ИНГИБИРОВАНИЯ ПРИРОДНОГО АНТИСМЫСЛОВОГО ТРАНСКРИПТА К р63
KR101838305B1 (ko) 2009-12-29 2018-03-13 큐알엔에이, 인크. ＮＲＦ1(Ｎｕｃｌｅａｒ Ｒｅｓｐｉｒａｔｏｒｙ Ｆａｃｔｏｒ 1)에 대한 천연 안티센스 전사체의 억제에 의한 핵 호흡 인자 1 관련된 질환의 치료
NO2521784T3 (es) 2010-01-04 2018-05-05
JP5963680B2 (ja) 2010-01-06 2016-08-03 カッパーアールエヌエー，インコーポレイテッド 膵臓発生遺伝子に対する天然アンチセンス転写物の阻害による膵臓発生遺伝子疾患の治療
US9200277B2 (en) 2010-01-11 2015-12-01 Curna, Inc. Treatment of sex hormone binding globulin (SHBG) related diseases by inhibition of natural antisense transcript to SHBG
EP2525808A2 (en) 2010-01-19 2012-11-28 The Trustees Of Columbia University In The City Of New York Osteocalcin as a treatment for male reproductive disorders
EA031322B1 (ru) 2010-01-22 2018-12-28 Дау Агросайенсиз Ллс Клетка или клеточная линия для экспрессии экзогенных нуклеотидных последовательностей и применение клетки или клеточной линии
DK2529015T3 (en) 2010-01-25 2018-02-26 Curna Inc TREATMENT OF RNASE H1-RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO RNASE H1
PT2534173T (pt) 2010-02-08 2019-10-31 Sangamo Therapeutics Inc Semidomínios de clivagem manipulados
CA2788850C (en) 2010-02-09 2019-06-25 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
CN102844435B (zh) 2010-02-22 2017-05-10 库尔纳公司 通过抑制吡咯啉‑5‑羧酸还原酶1(pycr1)的天然反义转录物而治疗pycr1相关疾病
AR080243A1 (es) 2010-02-23 2012-03-21 Genentech Inc Composiciones y metodos para el diagnostico y tratamiento de tumores
WO2011109427A2 (en) 2010-03-01 2011-09-09 Alnylam Pharmaceuticals, Inc. Improving the biological activity of sirna through modulation of its thermodynamic profile
EP3231872B1 (en) 2010-03-08 2020-05-06 Monsanto Technology LLC Polynucleotide molecules for gene regulation in plants
AU2011227050B2 (en) 2010-03-19 2016-12-08 University Of South Alabama Methods and compositions for the treatment of cancer
CN103038343A (zh) 2010-03-23 2013-04-10 英特瑞克斯顿股份有限公司 条件表达治疗蛋白的载体，包含所述载体的宿主细胞，及其应用
IL265674B2 (en) 2010-03-24 2024-05-01 Phio Pharm Corp Rana disorder in cutaneous and fibrotic symptoms
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
US9247720B2 (en) 2010-03-24 2016-02-02 Institut National de la Santé et de la Recherche Médicale Primate model from the family cercopithecidae infected by a HBV strain of human genotype
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
WO2011119920A2 (en) 2010-03-25 2011-09-29 Oregon Health & Science University Cmv glycoproteins and recombinant vectors
WO2011120023A1 (en) 2010-03-26 2011-09-29 Marina Biotech, Inc. Nucleic acid compounds for inhibiting survivin gene expression uses thereof
US8883218B2 (en) 2010-03-26 2014-11-11 The Board Of Trustees Of The University Of Arkansas Anti-cancer nanoparticle compositions and methods of use
JP5978203B2 (ja) 2010-04-09 2016-08-24 カッパーアールエヌエー，インコーポレイテッド Ｆｇｆ２１に対する天然アンチセンス転写物の阻害による線維芽細胞増殖因子（ｆｇｆ２１）線維芽細胞増殖因子ｆｇｆ２１）関連疾患の治療
US8790621B2 (en) * 2010-04-12 2014-07-29 Iowa State University Research Foundation, Inc. Nanoparticles and nanoparticle compositions
CA2796459C (en) 2010-04-16 2016-05-24 Salk Institute For Biological Studies Methods for treating metabolic disorders using fgf-1
WO2011133584A2 (en) 2010-04-19 2011-10-27 Marina Biotech, Inc. Nucleic acid compounds for inhibiting hras gene expression and uses thereof
JP5902616B2 (ja) 2010-04-28 2016-04-13 協和発酵キリン株式会社 カチオン性脂質
WO2011139842A2 (en) 2010-04-28 2011-11-10 Marina Biotech, Inc. Nucleic acid compounds for inhibiting fgfr3 gene expression and uses thereof
JP5902617B2 (ja) 2010-04-28 2016-04-13 協和発酵キリン株式会社 カチオン性脂質
PT2563920T (pt) 2010-04-29 2017-05-26 Ionis Pharmaceuticals Inc Modulação da expressão de transtirretina
KR20130079384A (ko) 2010-05-03 2013-07-10 제넨테크, 인크. 종양의 진단 및 치료를 위한 조성물 및 방법
CN107988228B (zh) 2010-05-03 2022-01-25 库尔纳公司 通过抑制沉默调节蛋白(sirt)的天然反义转录物而治疗沉默调节蛋白(sirt)相关疾病
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US9457079B2 (en) 2010-05-12 2016-10-04 The Trustees Of Columbia University In The City Of New York Methods for producing enteroendocrine cells that make and secrete insulin
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
TWI531370B (zh) 2010-05-14 2016-05-01 可娜公司 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
US8658780B2 (en) 2010-05-18 2014-02-25 California Institute Of Technology Triggered covalent probes for imaging and silencing genetic expression
CN102947451B (zh) 2010-05-26 2017-09-22 库尔纳公司 通过抑制无调同源物1(atoh1)的天然反义转录物而治疗atoh1相关疾病
EP2580238A1 (en) 2010-06-09 2013-04-17 Zymogenetics, Inc. Dimeric vstm3 fusion proteins and related compositions and methods
AU2011268146A1 (en) 2010-06-17 2013-01-10 Actogenix Nv Compositions and methods for treating inflammatory conditions
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012006378A1 (en) 2010-07-06 2012-01-12 Novartis Ag Liposomes with lipids having an advantageous pka- value for rna delivery
ES2649896T3 (es) 2010-07-06 2018-01-16 Glaxosmithkline Biologicals Sa Emulsiones catiónicas de aceite en agua
LT3243526T (lt) 2010-07-06 2020-02-10 Glaxosmithkline Biologicals S.A. Rnr pristatymas, skirtas keleto imuninio atsako paleidimui
DK2591114T3 (en) 2010-07-06 2016-08-29 Glaxosmithkline Biologicals Sa Immunization of large mammals with low doses of RNA
DK2593547T3 (en) 2010-07-14 2018-02-26 Curna Inc Treatment of Discs large homolog (DLG) related diseases by inhibition of natural antisense transcript to DLG
US8877438B2 (en) 2010-07-20 2014-11-04 California Institute Of Technology Self-assembled polynucleotide structure
US9834439B2 (en) 2010-07-20 2017-12-05 California Institute Of Technology Biomolecular self-assembly
US8962241B2 (en) 2010-07-20 2015-02-24 California Institute Of Technology Triggered molecular geometry based bioimaging probes
WO2012015938A2 (en) 2010-07-27 2012-02-02 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
AU2011294776B2 (en) 2010-08-23 2016-02-04 Wyeth Llc Stable formulations of Neisseria meningitidis rLP2086 antigens
DK2611461T3 (da) 2010-08-31 2022-05-16 Glaxosmithkline Biologicals Sa Pegylerede liposomer til afgivelse af RNA, der koder immunogen
CA2809758C (en) 2010-09-10 2021-07-13 Wyeth Llc Non-lipidated variants of neisseria meningitidis orf2086 antigens
EP2616431B1 (en) 2010-09-14 2016-11-09 Council of Scientific & Industrial Research Novel cationic amphiphiles with mannose-mimicking head-groups and a process for the preparation thereof
EP2622090B1 (en) 2010-09-27 2019-06-19 Sangamo Therapeutics, Inc. Compositions for inhibiting viral entry into cells
CA2813738A1 (en) 2010-10-05 2012-04-12 Genentech, Inc. Mutant smoothened and methods of using the same
CA2813901C (en) 2010-10-06 2019-11-12 Curna, Inc. Treatment of sialidase 4 (neu4) related diseases by inhibition of natural antisense transcript to neu4
WO2012051211A2 (en) 2010-10-11 2012-04-19 Novartis Ag Antigen delivery platforms
CA2815212A1 (en) 2010-10-22 2012-04-26 Curna, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
DK2633052T3 (en) 2010-10-27 2018-07-16 Curna Inc TREATMENT OF INTERFERON-RELATED DEVELOPMENT REGULATOR 1 (IFRD1) -RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPT TO IFRD1
US20140134181A1 (en) 2010-11-05 2014-05-15 Kenneth E. Lipson Treatment Method For Lung Remodeling Diseases
US8987225B2 (en) 2010-11-23 2015-03-24 Curna, Inc. Treatment of NANOG related diseases by inhibition of natural antisense transcript to NANOG
CA2819635A1 (en) 2010-12-01 2012-06-07 Spinal Modulation, Inc. Directed delivery of agents to neural anatomy
EP2648706A4 (en) 2010-12-06 2014-07-23 Penn State Res Found COMPOSITIONS AND METHODS RELATING TO PROLIFERATIVE DISEASES
EP2649178B8 (en) 2010-12-09 2017-08-30 Institut Pasteur Mgmt-based method for obtaining high yield of recombinant protein expression
US9095598B2 (en) 2010-12-28 2015-08-04 The Board Of Trustees Of The University Of Arkansas Stilbenoid derivatives and their uses
US9428565B2 (en) 2011-01-31 2016-08-30 The General Hospital Corporation Treatment and bioluminescent visualization using multimodal TRAIL molecules
JP6177692B2 (ja) 2011-02-02 2017-08-09 エクスカリアード・ファーマシューティカルズ，インク 結合組織増殖因子（ｃｔｇｆ）をターゲティングするアンチセンス化合物を用いた、ケロイドまたは肥厚性瘢痕の治療法
WO2012109495A1 (en) 2011-02-09 2012-08-16 Metabolic Solutions Development Company, Llc Cellular targets of thiazolidinediones
EP2492279A1 (en) 2011-02-25 2012-08-29 Laboratorios Del. Dr. Esteve, S.A. Rapid immunogen selection method using lentiviral display
CA2828411A1 (en) 2011-03-04 2012-09-13 Intrexon Corporation Vectors conditionally expressing protein
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
WO2012135696A2 (en) 2011-04-01 2012-10-04 University Of South Alabama Methods and compositions for the diagnosis, classification, and treatment of cancer
AU2012242642A1 (en) 2011-04-13 2013-05-02 Ionis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US20130101628A1 (en) * 2011-04-29 2013-04-25 Northwestern University Novel vegf mimetic peptide-based scaffolds for therapeutic angiogenesis and methods for their use
PL2709981T3 (pl) * 2011-05-20 2019-07-31 Merck Patent Gmbh Otrzymywanie stabilnych modyfikacji krystalicznych chlorku dotap
NZ730355A (en) 2011-05-24 2022-10-28 Tron Translationale Onkologie An Der Univ Der Johannes Gutenberg Univ Mainz Gemeinnuetzige Gmbh Individualized vaccines for cancer
CN111671918A (zh) 2011-06-08 2020-09-18 川斯勒佰尔公司 Mrna递送的脂质纳米颗粒组合物和方法
KR102043422B1 (ko) 2011-06-09 2019-11-11 큐알엔에이, 인크. 프라탁신 (fxn)에 대한 자연 안티센스 전사체의 저해에 의한 프라탁신 (fxn) 관련된 질환의 치료
DK2691530T3 (en) 2011-06-10 2018-05-22 Univ Oregon Health & Science CMV GLYCOPROTEIN AND RECOMBINANT VECTORS
EP2721156B1 (en) 2011-06-16 2016-12-21 Ionis Pharmaceuticals, Inc. Antisense modulation of fibroblast growth factor receptor 4 expression
SG10201913683WA (en) 2011-06-21 2020-03-30 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
CA2840965C (en) 2011-07-06 2021-03-02 Novartis Ag Cationic oil-in-water emulsions
EP2729125B1 (en) 2011-07-06 2017-12-13 GlaxoSmithKline Biologicals SA Oil-in-water emulsions that contain nucleic acids
ES2656050T3 (es) 2011-07-06 2018-02-22 Glaxosmithkline Biologicals Sa Composiciones de combinación inmunogénica y usos de las mismas
EP2546358A1 (en) 2011-07-15 2013-01-16 Laboratorios Del. Dr. Esteve, S.A. Methods and reagents for efficient control of HIV progression
JP6214530B2 (ja) 2011-07-15 2017-10-18 ザ ジェネラル ホスピタル コーポレイション 転写活性化因子様エフェクターの組立て方法
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
AU2012216792A1 (en) 2011-09-12 2013-03-28 International Aids Vaccine Initiative Immunoselection of recombinant vesicular stomatitis virus expressing HIV-1 proteins by broadly neutralizing antibodies
US10829828B2 (en) 2011-09-13 2020-11-10 Monsanto Technology Llc Methods and compositions for weed control
MX350771B (es) 2011-09-13 2017-09-15 Monsanto Technology Llc Métodos y composiciones para el control de malezas.
US10760086B2 (en) 2011-09-13 2020-09-01 Monsanto Technology Llc Methods and compositions for weed control
US10806146B2 (en) 2011-09-13 2020-10-20 Monsanto Technology Llc Methods and compositions for weed control
DK2758433T3 (en) 2011-09-19 2018-01-15 Axon Neuroscience Se PROTEIN-BASED THERAPY AND DIAGNOSIS OF TAU-MEDIATED PATHOLOGY OF ALZHEIMER'S DISEASE
EP2760463B1 (en) 2011-09-20 2018-11-21 The University of North Carolina At Chapel Hill Regulation of sodium channels by plunc proteins
AU2012312433B2 (en) 2011-09-20 2017-07-06 Ionis Pharmaceuticals, Inc. Antisense modulation of GCGR expression
US20130071403A1 (en) 2011-09-20 2013-03-21 Vical Incorporated Synergistic anti-tumor efficacy using alloantigen combination immunotherapy
ES2961613T3 (es) 2011-09-21 2024-03-12 Sangamo Therapeutics Inc Métodos y composiciones para la regulación de la expresión transgénica
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
WO2013053765A1 (en) 2011-10-11 2013-04-18 Proyecto De Biomedicina Cima, S.L. A non-human animal model of mucosa-associated lymphoid tissue (malt) lymphoma
KR20140084232A (ko) 2011-10-25 2014-07-04 아이시스 파마수티컬즈 인코포레이티드 Gccr 발현의 안티센스 조절
US9402894B2 (en) 2011-10-27 2016-08-02 International Aids Vaccine Initiative Viral particles derived from an enveloped virus
JP2013095755A (ja) 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd カチオン性脂質
EA201490993A1 (ru) 2011-11-18 2014-09-30 Элнилэм Фармасьютикалз, Инк. МОДИФИЦИРОВАННЫЕ СРЕДСТВА РНКи
WO2013075035A1 (en) 2011-11-18 2013-05-23 Alnylam Pharmaceuticals Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
US9463247B2 (en) * 2011-12-07 2016-10-11 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
CA3165769A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
MX349562B (es) 2011-12-09 2017-08-02 Pasteur Institut Ensayo de inmuno deteccion multiplex.
US20140045913A1 (en) 2011-12-12 2014-02-13 Kyowa Hakko Kirin Co., Ltd. Lipid nano particles comprising combination of cationic lipid
EP2792367A4 (en) 2011-12-12 2015-09-30 Kyowa Hakko Kirin Co Ltd LIPID NANOPARTICLES FOR A DRUG DELIVERY SYSTEM CONTAINING CATIONIC LIPIDS
JP2015501844A (ja) 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
US9073960B2 (en) 2011-12-22 2015-07-07 Alios Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
WO2013103401A1 (en) 2012-01-06 2013-07-11 University Of South Alabama Methods and compositions for the treatment of cancer
EP2802338B1 (en) 2012-01-09 2018-03-07 Serpin Pharma, LLC Peptides and methods of using same
JP6275655B2 (ja) 2012-02-24 2018-02-07 プロティバ バイオセラピューティクス インコーポレイテッド トリアルキルカチオン性脂質およびその使用方法
KR102084539B1 (ko) 2012-02-29 2020-03-04 상가모 테라퓨틱스, 인코포레이티드 헌팅턴병을 치료하기 위한 방법 및 조성물
SA115360586B1 (ar) 2012-03-09 2017-04-12 فايزر انك تركيبات لعلاج الالتهاب السحائي البكتيري وطرق لتحضيرها
EP4043029A1 (en) 2012-03-09 2022-08-17 Pfizer Inc. Neisseria meningitidis compositions and methods thereof
CN110438125A (zh) 2012-03-15 2019-11-12 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
US9441007B2 (en) 2012-03-21 2016-09-13 Alios Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
USRE48171E1 (en) 2012-03-21 2020-08-25 Janssen Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
EP2833923A4 (en) 2012-04-02 2016-02-24 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF PROTEINS
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
WO2013162748A1 (en) 2012-04-27 2013-10-31 The Trustees Of The University Of Pennsylvania Anti-tumor endothelial marker-1 (tem1) antibody variants and uses thereof
HRP20220158T1 (hr) 2012-05-21 2022-04-15 Insmed Incorporated Sustavi za liječenje plućnih infekcija
CN104619843B (zh) 2012-05-24 2020-03-06 A.B.种子有限公司 用于使基因表达沉默的组合物和方法
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
HRP20211424T2 (hr) * 2012-06-08 2022-02-18 Nitto Denko Corporation Lipidi za formulacije namijenjene unosu terapijskog sredstva
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
ES2631608T3 (es) 2012-06-27 2017-09-01 International Aids Vaccine Initiative Variante de la glicoproteína Env del VIH-1
TW201726600A (zh) 2012-07-06 2017-08-01 協和醱酵麒麟有限公司 陽離子性脂質
EP2872154B1 (en) 2012-07-11 2017-05-31 Sangamo BioSciences, Inc. Methods and compositions for delivery of biologics
HUE051612T2 (hu) 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
TW201408324A (zh) 2012-07-16 2014-03-01 Kyowa Hakko Kirin Co Ltd ＫＲＡＳ基因表現抑制ＲＮＡｉ醫藥組合物
CA2879683C (en) 2012-08-03 2023-02-14 Alnylam Pharmaceuticals, Inc. Modified rnai agents
EP2698377A1 (en) 2012-08-17 2014-02-19 Laboratorios Del. Dr. Esteve, S.A. Enhanced rapid immunogen selection method for HIV gp120 variants
KR102474010B1 (ko) 2012-08-29 2022-12-02 상가모 테라퓨틱스, 인코포레이티드 유전적 병태를 치료하기 위한 방법 및 조성물
EP2890720B1 (en) 2012-08-30 2019-07-17 The General Hospital Corporation Compositions and methods for treating cancer
US9365856B2 (en) 2012-09-14 2016-06-14 The Board Of Trustees Of The University Of Arkansas Methods of using a serum response factor isoform
EP2943194A1 (en) 2012-09-17 2015-11-18 Chemedest Ltd. Treatment of peripheral neuropathy using gfr(alpha)3 type receptor agonists
EP2897639A4 (en) 2012-09-21 2016-05-04 Frank Bedu-Addo IMPROVED VACCINE COMPOSITIONS AND METHODS OF USE
DK2906684T3 (da) 2012-10-10 2020-09-28 Sangamo Therapeutics Inc T-celle-modificerende forbindelser og anvendelser deraf
EP3789405A1 (en) 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
WO2014082729A1 (en) 2012-11-28 2014-06-05 Biontech Ag Individualized vaccines for cancer
CN104884047A (zh) 2012-11-29 2015-09-02 英斯梅德股份有限公司 稳定的万古霉素制剂
PL2929031T4 (pl) 2012-12-05 2022-06-27 Alnylam Pharmaceuticals, Inc. Kompozycje iRNA PCSK9 i sposoby ich zastosowania
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
MX2015007549A (es) 2012-12-12 2017-01-20 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
PL2896697T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Projektowanie systemów, sposoby i optymalizowane kompozycje kierujące do manipulacji sekwencją
PL2898075T3 (pl) 2012-12-12 2016-09-30 PROJEKTOWANIE i OPTYMALIZACJA ULEPSZONYCH SYSTEMÓW, SPOSOBY I KOMPOZYCJE ENZYMÓW DO MANIPULACJI SEKWENCJĄ
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
US8993233B2 (en) 2012-12-12 2015-03-31 The Broad Institute Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2825654B1 (en) 2012-12-12 2017-04-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
AU2013371825B2 (en) 2013-01-01 2019-10-24 A.B. Seeds Ltd. Methods of introducing dsRNA to plant seeds for modulating gene expression
US10683505B2 (en) 2013-01-01 2020-06-16 Monsanto Technology Llc Methods of introducing dsRNA to plant seeds for modulating gene expression
WO2014107739A1 (en) 2013-01-07 2014-07-10 Eleven Biotherapeutics, Inc. Antibodies against pcsk9
EP3919505B1 (en) 2013-01-16 2023-08-30 Emory University Uses of cas9-nucleic acid complexes
WO2014124284A1 (en) 2013-02-07 2014-08-14 The General Hospital Corporation Tale transcriptional activators
US9790269B2 (en) 2013-02-08 2017-10-17 Misfolding Diagnostics, Inc. Transthyretin antibodies and uses thereof
US9371352B2 (en) 2013-02-08 2016-06-21 Vaccinex, Inc. Modified glycolipids and methods of making and using the same
US10017581B2 (en) 2013-02-20 2018-07-10 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
ES2685894T3 (es) 2013-03-08 2018-10-15 Pfizer Inc. Polipéptidos de fusión inmunogénicos
WO2014164703A1 (en) 2013-03-11 2014-10-09 University Of Florida Research Foundation, Inc. Delivery of card protein as therapy for occular inflammation
KR20160029731A (ko) 2013-03-12 2016-03-15 더 제너럴 하스피탈 코포레이션 변형된 뮐러관 억제 물질(mis) 단백질 및 질환 치료를 위한 그 용도
UY35379A (es) 2013-03-13 2014-09-30 Monsanto Technology Llc ?métodos y composiciones para el control de malezas?.
AU2014248958A1 (en) 2013-03-13 2015-10-01 Monsanto Technology Llc Methods and compositions for weed control
US10501531B2 (en) 2013-03-13 2019-12-10 Prothena Biosciences Limited Tau immunotherapy
KR102605775B1 (ko) 2013-03-14 2023-11-29 알닐람 파마슈티칼스 인코포레이티드 보체 성분 Ｃ5 ｉＲＮＡ 조성물 및 그 이용 방법
JP2016518812A (ja) 2013-03-14 2016-06-30 ガラパゴス・ナムローゼ・フェンノートシャップＧａｌａｐａｇｏｓ Ｎ．Ｖ． 線維症治療において有用な分子標的及び前記標的のインヒビター
WO2014139885A2 (en) 2013-03-14 2014-09-18 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of diseases associated with epithelial mesenchymal transition
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US20160054304A1 (en) 2013-03-14 2016-02-25 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of fibrotic diseases
ES2708561T3 (es) 2013-03-14 2019-04-10 Translate Bio Inc Métodos para la purificación de ARN mensajero
AU2014236305B2 (en) 2013-03-14 2019-01-17 Ethris Gmbh CFTR mRNA compositions and related methods and uses
AU2013381866A1 (en) 2013-03-15 2015-09-24 The Penn State Research Foundation Compositions and methods including celecoxib and plumbagin relating to treatment of cancer
WO2014152497A2 (en) 2013-03-15 2014-09-25 The Trustees Of Columbia University In The City Of New York Osteocalcin as a treatment for cognitive disorders
WO2014144380A1 (en) 2013-03-15 2014-09-18 Intrexon Corporation Boron-containing diacylhydrazines
CA2905696A1 (en) 2013-03-15 2014-09-18 The Penn State Research Foundation Compositions and methods including leelamine and arachidonyl trifluoromethyl ketone relating to treatment of cancer
US10568328B2 (en) 2013-03-15 2020-02-25 Monsanto Technology Llc Methods and compositions for weed control
CN110237270A (zh) 2013-03-15 2019-09-17 洛马林达大学 自身免疫性疾病的治疗
WO2014180490A1 (en) 2013-05-10 2014-11-13 Biontech Ag Predicting immunogenicity of t cell epitopes
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
US20140349405A1 (en) 2013-05-22 2014-11-27 Wisconsin Alumni Research Foundation Rna-directed dna cleavage and gene editing by cas9 enzyme from neisseria meningitidis
CN110317810A (zh) 2013-05-22 2019-10-11 阿尔尼拉姆医药品有限公司 Tmprss6 irna组合物及其使用方法
KR20220154244A (ko) 2013-05-22 2022-11-21 알닐람 파마슈티칼스 인코포레이티드 ＳＥＲＰＩＮＡ1 ｉＲＮＡ 조성물 및 이의 사용 방법
US20160130585A1 (en) 2013-05-28 2016-05-12 The Johns Hopkins University Aptamers for the treatment of sickle cell disease
JP6869720B2 (ja) 2013-06-13 2021-05-12 アンチセンス セラピューティクス リミテッド 併用療法
EP3011029B1 (en) 2013-06-17 2019-12-11 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
WO2014204727A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
ES2767318T3 (es) 2013-06-17 2020-06-17 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas
KR20160034901A (ko) 2013-06-17 2016-03-30 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작에 최적화된 ｃｒｉｓｐｒ-ｃａｓ 이중 닉카아제 시스템, 방법 및 조성물
CN107995927B (zh) 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
US20160152952A1 (en) 2013-06-25 2016-06-02 Temple University-Of The Commonwealth System Of Higher Education Cortical Bone-Derived Stem Cells
US9856472B2 (en) 2013-07-01 2018-01-02 California Institute Of Technology Small conditional RNAs
US9850496B2 (en) 2013-07-19 2017-12-26 Monsanto Technology Llc Compositions and methods for controlling Leptinotarsa
EP3030663B1 (en) 2013-07-19 2019-09-04 Monsanto Technology LLC Compositions and methods for controlling leptinotarsa
CN110974981A (zh) 2013-07-23 2020-04-10 野草莓树生物制药公司 用于递送信使rna的组合物和方法
EP3024936B1 (en) 2013-07-25 2019-09-04 Exicure, Inc. Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
WO2015023797A1 (en) * 2013-08-13 2015-02-19 Northwestern University Lipophilic nanoparticles for drug delivery
WO2015031619A1 (en) 2013-08-28 2015-03-05 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
WO2015031645A1 (en) 2013-08-28 2015-03-05 Washington University Monoclonal antibodies to human 14-3-3 epsilon and human 14-3-3 epsilon sv
EP2848937A1 (en) 2013-09-05 2015-03-18 International Aids Vaccine Initiative Methods of identifying novel HIV-1 immunogens
WO2015035231A1 (en) 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
MX369534B (es) 2013-09-08 2019-11-11 Pfizer Composiciones de neisseria meningitidis y sus metodos.
EP3047023B1 (en) 2013-09-19 2019-09-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Compositions and methods for inhibiting jc virus (jcv)
AR097738A1 (es) 2013-09-23 2016-04-13 Alnylam Pharmaceuticals Inc Métodos para tratar o prevenir enfermedades asociadas con la transtiretina (ttr)
BR112016006192A2 (pt) 2013-09-25 2017-09-26 Zoetis Services Llc composição de vacina de divergente de pcv2b e métodos de uso
EP2873423B1 (en) 2013-10-07 2017-05-31 International Aids Vaccine Initiative Soluble hiv-1 envelope glycoprotein trimers
CN116836957A (zh) 2013-10-17 2023-10-03 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのｍＲＮＡ療法
CN106413811A (zh) 2013-10-22 2017-02-15 夏尔人类遗传性治疗公司 精氨基琥珀酸合成酶缺乏症的mrna疗法
NZ719544A (en) 2013-11-04 2022-09-30 Beeologics Inc Compositions and methods for controlling arthropod parasite and pest infestations
US9469650B2 (en) 2013-11-08 2016-10-18 Board Of Trustees Of The University Of Arkansas Melampomagnolide B derivatives
US9487536B2 (en) 2013-11-08 2016-11-08 Board Of Trustees Of The University Of Arkansas Melampomagnolide B derivatives
CN105934524A (zh) 2013-11-11 2016-09-07 桑格摩生物科学股份有限公司 用于治疗亨廷顿氏病的方法和组合物
PT3492593T (pt) 2013-11-13 2021-10-18 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
CA2932122C (en) 2013-12-03 2022-04-19 Northwestern University Liposomal particles, methods of making same and uses thereof
CN105960265A (zh) 2013-12-04 2016-09-21 阿克赛医药公司 利用经化学修饰的寡核苷酸处理伤口愈合的方法
EP3757116A1 (en) 2013-12-09 2020-12-30 Sangamo Therapeutics, Inc. Methods and compositions for genome engineering
UA119253C2 (uk) 2013-12-10 2019-05-27 Біолоджикс, Інк. Спосіб боротьби із вірусом у кліща varroa та у бджіл
WO2015089321A2 (en) 2013-12-11 2015-06-18 The General Hospital Corporation Use of mullerian inhibiting substance (mis) proteins for contraception and ovarian reserve preservation
AU2014362262B2 (en) 2013-12-12 2021-05-13 Alnylam Pharmaceuticals, Inc. Complement component iRNA compositions and methods of use thereof
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
KR20160089527A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのｃｒｉｓｐｒ−ｃａｓ系及び組成物の送達、使用及び治療適用
AU2014361784A1 (en) 2013-12-12 2016-06-23 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for HBV and viral diseases and disorders
WO2015106128A2 (en) 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
US10334848B2 (en) 2014-01-15 2019-07-02 Monsanto Technology Llc Methods and compositions for weed control using EPSPS polynucleotides
WO2015116902A1 (en) 2014-01-31 2015-08-06 Genentech, Inc. G-protein coupled receptors in hedgehog signaling
EP3102673B1 (en) 2014-02-03 2020-04-15 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a beta thalessemia
JP6736467B2 (ja) 2014-02-04 2020-08-05 ジェネンテック， インコーポレイテッド 平滑化変異体及びその使用方法
KR102389968B1 (ko) 2014-02-11 2022-04-25 알닐람 파마슈티칼스 인코포레이티드 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법
WO2015127094A1 (en) 2014-02-19 2015-08-27 University Of Florida Research Foundation, Inc. Delivery of nrf2 as therapy for protection against reactive oxygen species
EP3110401A4 (en) 2014-02-25 2017-10-25 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
KR101605421B1 (ko) 2014-03-05 2016-03-23 국립암센터 B 세포 림프종 세포를 특이적으로 인지하는 단일클론항체 및 이의 용도
ES2879373T3 (es) 2014-03-18 2021-11-22 Sangamo Therapeutics Inc Métodos y composiciones para la regulación de la expresión de proteínas de dedo de zinc
BR112016022711A2 (pt) 2014-04-01 2017-10-31 Monsanto Technology Llc composições e métodos para controle de pragas de inseto
ES2750661T3 (es) 2014-04-25 2020-03-26 Translate Bio Inc Métodos para la purificación de ARN mensajero
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
JP2017514908A (ja) 2014-05-01 2017-06-08 アールエックスアイ ファーマシューティカルズ コーポレーション 核酸分子を利用する目の前部における障害の処置のための方法
EP3708170A1 (en) 2014-05-05 2020-09-16 BioVentures, LLC Compositions and methods for inhibiting antiapoptotic bcl-2 proteins as anti-aging agents
WO2015171918A2 (en) 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
RU2691102C2 (ru) 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
CA2949078C (en) 2014-05-15 2022-09-20 Insmed Incorporated Methods for treating pulmonary non-tuberculous mycobacterial infections
EA201692370A1 (ru) 2014-05-22 2017-03-31 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ иРНК АНГИОТЕНЗИНОГЕНА (AGT) И СПОСОБЫ ИХ ИСПОЛЬЗОВАНИЯ
BR112016028023A2 (pt) 2014-05-30 2017-08-22 Univ Leland Stanford Junior Composições e métodos de administração de tratamentos para infecções virais latentes
CA2953216C (en) 2014-06-04 2020-12-22 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
EA039693B1 (ru) 2014-06-16 2022-02-28 Дзе Джонс Хопкинс Юниверсити Композиции и способы для экспрессии рнк-проводников crispr с использованием промотора h1
AU2015280252A1 (en) 2014-06-23 2017-01-12 Monsanto Technology Llc Compositions and methods for regulating gene expression via RNA interference
US11807857B2 (en) 2014-06-25 2023-11-07 Monsanto Technology Llc Methods and compositions for delivering nucleic acids to plant cells and regulating gene expression
WO2015200901A1 (en) 2014-06-26 2015-12-30 The Trustees Of Columbia University In The City Of New York Inhibition of serotonin expression in gut enteroendocrine cells results in conversion to insulin-positive cells
CN106572974B (zh) 2014-07-15 2021-04-23 生命技术公司 用于将分子有效递送到细胞的具有脂质聚集体的组合物和方法
EP3169773B1 (en) 2014-07-15 2023-07-12 Juno Therapeutics, Inc. Engineered cells for adoptive cell therapy
JP2017522388A (ja) 2014-07-22 2017-08-10 バイオベンチャーズ・リミテッド・ライアビリティ・カンパニーＢｉｏＶｅｎｔｕｒｅｓ， ＬＬＣ 老化細胞を選択的に枯渇させるための組成物及び方法
EP3172222A4 (en) 2014-07-24 2018-03-21 Washington University Compositions targeting radiation-induced molecules and methods of use thereof
KR102330593B1 (ko) 2014-07-28 2021-11-26 에스케이이노베이션 주식회사 신규 이소프렌 신타아제 및 이를 이용한 이소프렌의 제조방법
RU2021123470A (ru) 2014-07-29 2021-09-06 Монсанто Текнолоджи Ллс Композиции и способы борьбы с насекомыми-вредителями
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
DK3180426T3 (da) 2014-08-17 2020-03-30 Broad Inst Inc Genomredigering ved anvendelse af cas9-nickaser
SG10201903290YA (en) 2014-08-20 2019-05-30 Alnylam Pharmaceuticals Inc Modified double-stranded rna agents
WO2016033424A1 (en) 2014-08-29 2016-03-03 Genzyme Corporation Methods for the prevention and treatment of major adverse cardiovascular events using compounds that modulate apolipoprotein b
CN107073294A (zh) 2014-09-05 2017-08-18 阿克赛医药公司 使用靶向tyr或mmp1的核酸治疗老化和皮肤病症的方法
EP3939593A1 (en) 2014-09-10 2022-01-19 Washington University Compositions and methods for treatment of pre-cancerous skin lesions
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
PL3194570T3 (pl) 2014-09-16 2021-12-20 Sangamo Therapeutics, Inc. Sposoby i kompozycje do inżynierii genomowej w której pośredniczy nukleaza i korekty hematopoetycznych komórek macierzystych
CA2961738A1 (en) 2014-09-17 2016-03-24 Intrexon Corporation Boron-containing diacylhydrazine compounds
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016045732A1 (en) 2014-09-25 2016-03-31 Biontech Rna Pharmaceuticals Gmbh Stable formulations of lipids and liposomes
EP3201338B1 (en) 2014-10-02 2021-11-03 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis b virus gene expression
CR20170181A (es) 2014-10-06 2017-05-31 Exicure Inc Compuestos anti-tnf
US20170304459A1 (en) 2014-10-10 2017-10-26 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhalation delivery of conjugated oligonucleotide
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
WO2016064832A1 (en) 2014-10-20 2016-04-28 The Curators Of The University Of Missouri Method for treating lymphedema
EP3212794B1 (en) 2014-10-30 2021-04-07 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
AU2015350120B2 (en) 2014-11-17 2021-05-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
WO2016081728A1 (en) 2014-11-19 2016-05-26 The Trustees Of Columbia University In The City Of New York Osteocalcin as a treatment for frailty associated with aging
WO2016085986A1 (en) 2014-11-24 2016-06-02 Northwestern University High density lipoprptein nanoparticles for inflammation
WO2016090166A1 (en) 2014-12-03 2016-06-09 Board Of Trustees Of The University Of Arkansas Melampomagnolide b dimers
US9834502B2 (en) 2014-12-08 2017-12-05 Nof Corporation Method for producing cationic lipid
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
US10793613B2 (en) 2014-12-15 2020-10-06 Washington University Compositions and methods for targeted cytokine delivery
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
AU2015369725A1 (en) 2014-12-24 2017-06-29 Massachusetts Institute Of Technology CRISPR having or associated with destabilization domains
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
EP3256589B1 (en) 2015-01-22 2021-12-22 Monsanto Technology LLC Compositions and methods for controlling leptinotarsa
RS64527B1 (sr) 2015-01-28 2023-09-29 Caribou Biosciences Inc Hibridni polinukleotidi crispr dnk/rnk i postupci upotrebe
WO2016128060A1 (en) 2015-02-12 2016-08-18 Biontech Ag Predicting t cell epitopes useful for vaccination
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
RU2723045C2 (ru) 2015-02-19 2020-06-08 Пфайзер Инк. Композиции neisseria meningitidis и способы их получения
US10736845B2 (en) * 2015-03-03 2020-08-11 Cureport Inc. Dual loaded liposomal pharmaceutical formulations
CN107530283A (zh) 2015-03-03 2018-01-02 奎尔波特股份有限公司 组合脂质体药物制剂
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3069730A3 (en) 2015-03-20 2017-03-15 International Aids Vaccine Initiative Soluble hiv-1 envelope glycoprotein trimers
EP3072901A1 (en) 2015-03-23 2016-09-28 International Aids Vaccine Initiative Soluble hiv-1 envelope glycoprotein trimers
WO2016161446A1 (en) 2015-04-03 2016-10-06 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
US20180112006A1 (en) 2015-04-17 2018-04-26 The General Hospital Corporation Agents, systems and methods for treating cancer
CA2984154A1 (en) 2015-05-01 2016-11-10 Onl Therapeutics, Inc. Peptide compositions and methods of use
BR112017021967A2 (pt) 2015-05-06 2018-07-31 Alnylam Pharmaceuticals Inc composições do fator xii (fator hageman) (f12), kallikrein b, plasma (fator fletcher) 1 (klkb1), e kininogen 1 (kng1) irna e métodos de uso dos mesmos
CA2986021A1 (en) 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
US20180154007A1 (en) 2015-05-20 2018-06-07 Syracuse University Glycemic Control Using Intrinsic Factor Bound to A Vitamin B12 Conjugate of a Glucagon-Like Peptide-1 Receptor Agonist
US10716827B2 (en) 2015-05-20 2020-07-21 Syracuse University Compositions and methods for enhancing peptide stability against protease digestion
EP3303586A1 (en) 2015-05-29 2018-04-11 Juno Therapeutics, Inc. Composition and methods for regulating inhibitory interactions in genetically engineered cells
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
EP3302497A4 (en) 2015-06-01 2019-01-16 Sarepta Therapeutics, Inc. EXCLUSION OF EXON INDUCED PAT TECHNOLOGY ANTISENSE IN COLLAGEN TYPE VII
CN107750125A (zh) 2015-06-02 2018-03-02 孟山都技术有限公司 用于将多核苷酸递送至植物中的组合物和方法
WO2016196782A1 (en) 2015-06-03 2016-12-08 Monsanto Technology Llc Methods and compositions for introducing nucleic acids into plants
US20180245074A1 (en) 2015-06-04 2018-08-30 Protiva Biotherapeutics, Inc. Treating hepatitis b virus infection using crispr
TW201710503A (zh) 2015-06-12 2017-03-16 長庚醫療財團法人林口長庚紀念醫院 新穎多核苷酸、載體、醫藥組成物以及其用途
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205728A1 (en) 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
EP3436575A1 (en) 2015-06-18 2019-02-06 The Broad Institute Inc. Novel crispr enzymes and systems
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
WO2017007825A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
EP3862005A1 (en) 2015-07-06 2021-08-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
WO2017011519A1 (en) 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US10758612B2 (en) 2015-07-15 2020-09-01 Washington University Antibodies to tumor associated complex N-glycans with terminal GlcNAcβ residues and methods of use thereof
MA42895A (fr) 2015-07-15 2018-05-23 Juno Therapeutics Inc Cellules modifiées pour thérapie cellulaire adoptive
EP3329003A2 (en) 2015-07-29 2018-06-06 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis b virus gene expression
WO2017024317A2 (en) 2015-08-06 2017-02-09 Dana-Farber Cancer Institute, Inc. Methods to induce targeted protein degradation through bifunctional molecules
MX2018002090A (es) 2015-08-24 2018-09-12 Halo Bio Rnai Therapeutics Inc Nanoparticulas de polinucleótido para modulación de expresión génica y sus usos.
CN113429455A (zh) 2015-08-28 2021-09-24 赛品制药有限责任公司 用于疾病治疗的方法
PE20181131A1 (es) 2015-09-02 2018-07-17 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi PARA LIGANDO 1 DE MUERTE CELULAR PROGRAMADA 1 (PD-L1) Y METODOS DE USO DE LAS MISMAS
EP3350328A1 (en) 2015-09-14 2018-07-25 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
JP6933379B2 (ja) 2015-09-24 2021-09-08 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 転移を低減するための方法および組成物
WO2017058881A1 (en) 2015-09-28 2017-04-06 The Trustees Of Columbia University In The City Of New York Use of pentoxifylline with immune checkpoint-blockade therapies for the treatment of melanoma
WO2017059902A1 (en) * 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
CA3000917A1 (en) 2015-10-09 2017-04-13 Monsanto Technology Llc Rna-guided nucleases and uses thereof
EP3359668A4 (en) 2015-10-09 2019-06-05 Sarepta Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF
CA3001590A1 (en) 2015-10-10 2017-04-13 Intrexon Corporation Improved therapeutic control of proteolytically sensitive, destabilized forms of interleukin-12
US10456360B2 (en) 2015-10-16 2019-10-29 Ipsen Biopharm Ltd. Stabilizing camptothecin pharmaceutical compositions
US11021707B2 (en) 2015-10-19 2021-06-01 Phio Pharmaceuticals Corp. Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA
US20170211142A1 (en) 2015-10-22 2017-07-27 The Broad Institute, Inc. Novel crispr enzymes and systems
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and their uses
US20180319815A1 (en) 2015-11-05 2018-11-08 Board Of Trustees Of The University Of Arkansas Hetero-substituted cyclic lactone analogues and uses thereof
WO2017075670A1 (en) 2015-11-05 2017-05-11 Children's Hospital Los Angeles "mobilizing leukemia cells"
CA3005251A1 (en) 2015-11-13 2017-05-18 Pds Biotechnology Corporation Lipids as synthetic vectors to enhance antigen processing and presentation ex-vivo in dendritic cell therapy
JP7110108B2 (ja) 2015-12-09 2022-08-01 ジンガン メディスン（オーストラリア） プロプライアタリー リミティド 治療用免疫調節組成物
WO2017100542A1 (en) 2015-12-10 2017-06-15 Alnylam Pharmaceuticals, Inc. Sterol regulatory element binding protein (srebp) chaperone (scap) irna compositions and methods of use thereof
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
CA3008382A1 (en) 2015-12-18 2017-06-22 Sangamo Therapeutics, Inc. Targeted disruption of the mhc cell receptor
AU2016369490C1 (en) 2015-12-18 2021-12-23 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
SG10202008046UA (en) 2015-12-23 2020-09-29 Univ Queensland Technology Nucleic acid oligomers and uses therefor
WO2017111172A1 (ja) 2015-12-25 2017-06-29 協和発酵キリン株式会社 カチオン性脂質としての化合物
WO2017120589A1 (en) 2016-01-08 2017-07-13 Washington University Compositions comprising chemerin and methods of use thereof
BR112018014288A2 (pt) 2016-01-15 2018-12-18 Univ Minnesota métodos e composições para o tratamento de doença neurológica
JP2019509980A (ja) 2016-01-29 2019-04-11 バイオベンチャーズ・リミテッド・ライアビリティ・カンパニーＢｉｏＶｅｎｔｕｒｅｓ， ＬＬＣ メランポマグノリドｂのトリアゾール誘導体及びその使用方法
EP3411056A4 (en) 2016-02-02 2019-10-02 Sangamo Therapeutics, Inc. COMPOUNDS FOR NETWORKING DNA BINDING DOMAINS AND SPLITTING DOMAINS
JP2019509721A (ja) 2016-02-04 2019-04-11 キュリス，インコーポレイテッド 突然変異体スムースンド及びその使用方法
AU2017213659B2 (en) 2016-02-05 2024-04-18 Washington University Compositions and methods for targeted cytokine delivery
ES2754785T3 (es) 2016-02-22 2020-04-20 Caribou Biosciences Inc Procedimientos de modulación de resultados de reparación de ADN
GB201604235D0 (en) 2016-03-11 2016-04-27 Ucl Business Plc Lipids and complexes for the delivery of biologically-active material to cells
CN109071609A (zh) 2016-03-24 2018-12-21 马凯特大学 定量鞭毛荧光标志物和标准品
US10213448B2 (en) 2016-03-25 2019-02-26 Novazoi Theranostics Ethanolamine-based lipid biosynthetic compounds, method of making and use thereof
CA3021267A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
EP3445856A1 (en) 2016-04-19 2019-02-27 The Broad Institute Inc. Novel crispr enzymes and systems
CA3026055A1 (en) 2016-04-19 2017-10-26 The Broad Institute, Inc. Novel crispr enzymes and systems
AU2017253107B2 (en) 2016-04-19 2023-07-20 Massachusetts Institute Of Technology CPF1 complexes with reduced indel activity
CN109152933B (zh) 2016-04-21 2022-12-02 生物风险投资有限责任公司 诱导抗细胞凋亡bcl-2家族蛋白的降解的化合物及其用途
US10752679B2 (en) 2016-05-02 2020-08-25 Prothena Biosciences Limited Tau immunotherapy
WO2017191561A1 (en) 2016-05-02 2017-11-09 Prothena Biosciences Limited Antibodies recognizing tau
PE20190261A1 (es) 2016-05-02 2019-02-25 Prothena Biosciences Ltd Anticuerpos que reconocen tau
KR102617833B1 (ko) 2016-05-06 2023-12-27 엑시큐어 오퍼레이팅 컴퍼니 인터류킨 17 수용체 mRNA의 특이적 녹다운을 위한 안티센스 올리고뉴클레오티드 (ASO)를 제시하는 리포좀성 구형 핵산 (SNA) 구축물
JP2019518028A (ja) 2016-06-10 2019-06-27 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 補体成分Ｃ５ｉＲＮＡ組成物及び発作性夜間血色素尿症（ＰＮＨ）を処置するためのその使用方法
CA3028074A1 (en) 2016-06-16 2018-12-20 Oslo Universitetssykehus Hf Improved gene editing
KR20230156150A (ko) 2016-06-17 2023-11-13 더 브로드 인스티튜트, 인코퍼레이티드 제vi형 crispr 오솔로그 및 시스템
WO2018005873A1 (en) 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
CN109563511A (zh) 2016-06-30 2019-04-02 阿布特斯生物制药公司 用于递送信使rna的组合物和方法
AU2017291727B2 (en) 2016-07-05 2021-07-08 California Institute Of Technology Fractional initiator hybridization chain reaction
US11766488B2 (en) 2016-07-05 2023-09-26 The Johns Hopkins University Compositions and methods comprising improvements of CRISPR guide RNAS using the H1 promoter
AU2017295720B2 (en) 2016-07-13 2021-07-22 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
CA3030587A1 (en) 2016-07-15 2018-01-18 Salk Institute For Biological Studies Methods and compositions for genome editing in non-dividing cells
CA2937157A1 (en) 2016-07-25 2018-01-25 Ucl Business Plc Protein-based t-cell receptor knockdown
CA3032699A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
US11878060B2 (en) 2016-08-07 2024-01-23 Novartis Ag mRNA-mediated immunization methods
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US20200283743A1 (en) 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
CN110114461A (zh) 2016-08-17 2019-08-09 博德研究所 新型crispr酶和系统
JP7203014B2 (ja) 2016-08-24 2023-01-12 サンガモ セラピューティクス， インコーポレイテッド 工学操作されたヌクレアーゼを使用した遺伝子発現の調節
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110418841A (zh) 2016-08-24 2019-11-05 桑格摩生物治疗股份有限公司 工程化的靶特异性核酸酶
IL264831B (en) 2016-08-30 2022-09-01 California Inst Of Techn Immunohistochemistry by hybridization chain reaction
US20210284998A1 (en) * 2016-10-03 2021-09-16 Precision Nanosystems Inc. Compositions for Transfecting Resistant Cell Types
US10961505B2 (en) 2016-10-05 2021-03-30 FUJIFILM Cellular Dynamics, Inc. Generating mature lineages from induced pluripotent stem cells with MECP2 disruption
AU2017343780B2 (en) 2016-10-13 2023-08-31 Juno Therapeutics, Inc. Immunotherapy methods and compositions involving tryptophan metabolic pathway modulators
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
US11219695B2 (en) 2016-10-20 2022-01-11 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
US20190314486A1 (en) 2016-10-21 2019-10-17 Merck Sharp & Dohme Corp. Influenza hemagglutinin protein vaccines
US11058653B2 (en) 2016-10-26 2021-07-13 Washington University Compositions comprising desaminotyrosine and uses thereof to enhance type I interferon stimulation
CA3041668A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
MX2019005235A (es) 2016-11-09 2019-12-05 Intrexon Corp Constructos de expresion de frataxina.
US10011848B2 (en) 2016-11-09 2018-07-03 City University Of Hong Kong System and method for delivery of substance into mammalian cells
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
WO2018102612A1 (en) 2016-12-02 2018-06-07 Juno Therapeutics, Inc. Engineered b cells and related compositions and methods
ES2968892T3 (es) 2016-12-08 2024-05-14 Univ Case Western Reserve Métodos y composiciones para aumentar la producción de mielina funcional
SG10201913552UA (en) 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
EP3559230B1 (en) 2016-12-20 2023-09-06 Bristol-Myers Squibb Company Methods for increasing the efficiency of homology directed repair (hdr) in the cellular genome
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
MX2019009011A (es) 2017-01-31 2019-09-26 Pfizer Composiciones de neisseria meningitidis y metodos respectivos.
WO2018148595A1 (en) 2017-02-10 2018-08-16 Washington University Antibodies to tip1 and methods of use thereof
WO2018157154A2 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Novel codon-optimized cftr mrna
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
US11795192B2 (en) 2017-03-10 2023-10-24 Lakewood Amedex, Inc. Antimicrobial compounds, compositions, and uses thereof
EP4361261A3 (en) 2017-03-15 2024-07-10 The Broad Institute Inc. Novel cas13b orthologues crispr enzymes and systems
IL269458B2 (en) 2017-03-23 2024-02-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
WO2018183403A1 (en) 2017-03-28 2018-10-04 Caribou Biosciences, Inc. Crispr-associated (cas) protein
KR20200006054A (ko) 2017-04-12 2020-01-17 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
SG11201909572QA (en) 2017-04-18 2019-11-28 Alnylam Pharmaceuticals Inc Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
US11351227B2 (en) 2017-04-27 2022-06-07 Washington University Chemokine decoy receptors of rodent gammaherpesviruses and uses thereof
US11596655B2 (en) 2017-04-27 2023-03-07 Washington University Activation of natural cytotoxicity receptor 2 (NCR2)
US11696954B2 (en) 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
US11932867B2 (en) 2017-04-28 2024-03-19 National Jewish Health Methods of treating rheumatoid arthritis using RNA-guided genome editing of HLA gene
PE20200695A1 (es) 2017-05-02 2020-06-16 Prothena Biosciences Ltd Anticuerpos que reconocen tau
WO2018206732A1 (en) 2017-05-09 2018-11-15 Vib Vzw Means and methods for treating bacterial infections
US20210278416A1 (en) 2017-05-09 2021-09-09 The Broad Institute, Inc. Gut microbiome function predicts response to anti-integrin biologic therapy in inflammatory bowel diseases
US11013905B2 (en) 2017-05-11 2021-05-25 Tci Llc Thermal interconnect for implantable blood pump
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
US10994025B2 (en) 2017-05-12 2021-05-04 Massachusetts Institute Of Technology Argonaute protein-double stranded RNA complexes and uses related thereto
EP3624824B1 (en) 2017-05-16 2024-07-10 Translate Bio, Inc. Codon-optimized mrna encoding cftr for use in treating cystic fibrosis
CA3067164A1 (en) 2017-06-12 2018-12-20 Washington University Zika virus strains for treatment of glioblastoma
BR112019027133B8 (pt) 2017-06-20 2022-08-23 Inst Curie Uso de uma célula imune modificada deficiente para suv39h1
RU2769475C2 (ru) 2017-06-23 2022-04-01 Инскрипта, Инк. Направляемые нуклеиновыми кислотами нуклеазы
JP7454494B2 (ja) 2017-06-26 2024-03-22 ザ・ブロード・インスティテュート・インコーポレイテッド 標的化された核酸編集のためのｃｒｉｓｐｒ／ｃａｓ－アデニンデアミナーゼ系の組成物、系及び方法
KR20200028997A (ko) 2017-07-13 2020-03-17 노오쓰웨스턴 유니버시티 올리고뉴클레오타이드-작용화된 금속-유기 프레임워크 나노입자를 제조하는 일반적이고 직접적인 방법
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
CA3108048A1 (en) 2017-07-31 2019-02-07 Washington University Pirfenidone derivatives for modulation of b lymphocyte activity and organ protection
WO2019036509A1 (en) 2017-08-14 2019-02-21 Washington University METHODS AND COMPOSITIONS FOR TREATING DISEASES ASSOCIATED WITH CANCER, INFLAMMATION OR IMMUNE RESPONSE
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US11097018B2 (en) 2017-09-06 2021-08-24 Washington University Organomedicinals for imaging and treatment of neurodegenerative disorders
WO2019055460A1 (en) 2017-09-13 2019-03-21 The Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR THE TREATMENT OF TRANSPOSON-ASSOCIATED DISEASES
CA3073848A1 (en) 2017-09-21 2019-03-28 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
JP7394753B2 (ja) 2017-10-18 2023-12-08 サレプタ セラピューティクス， インコーポレイテッド アンチセンスオリゴマー化合物
US11851679B2 (en) 2017-11-01 2023-12-26 Juno Therapeutics, Inc. Method of assessing activity of recombinant antigen receptors
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
CN107827762B (zh) * 2017-11-14 2020-10-02 苏州东南药业股份有限公司 一种(2,3-二油酰基-丙基)三甲基氯化铵及其制备方法和用途
US20200277573A1 (en) 2017-11-17 2020-09-03 Iovance Biotherapeutics, Inc. Til expansion from fine needle aspirates and small biopsies
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
JP2021503885A (ja) 2017-11-22 2021-02-15 アイオバンス バイオセラピューティクス，インコーポレイテッド 末梢血からの末梢血リンパ球（ｐｂｌ）の拡大培養
TW201936201A (zh) 2017-12-14 2019-09-16 美商堅固生物科技公司 基因之非病毒生產及遞送
EP3728281A1 (en) 2017-12-21 2020-10-28 Alnylam Pharmaceuticals Inc. Chirally-enriched double-stranded rna agents
CN111836887A (zh) 2018-01-08 2020-10-27 艾欧凡斯生物治疗公司 产生富含肿瘤抗原特异性t细胞的til产品的方法
US11713446B2 (en) 2018-01-08 2023-08-01 Iovance Biotherapeutics, Inc. Processes for generating TIL products enriched for tumor antigen-specific T-cells
WO2019136459A1 (en) 2018-01-08 2019-07-11 Iovance Biotherapeutics, Inc. Processes for generating til products enriched for tumor antigen-specific t-cells
WO2019143675A1 (en) 2018-01-17 2019-07-25 Vertex Pharmaceuticals Incorporated Dna-pk inhibitors
WO2019143677A1 (en) 2018-01-17 2019-07-25 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
US12005127B2 (en) 2018-01-17 2024-06-11 Vertex Pharmaceuticals Incorporated DNA-PK inhibitors
CA3088253A1 (en) 2018-01-22 2019-07-25 Bioventures, Llc Bcl-2 proteins degraders for cancer treatment
CN111655715A (zh) 2018-01-29 2020-09-11 默沙东公司 稳定化的rsv f蛋白及其用途
CN108129338A (zh) * 2018-02-01 2018-06-08 爱斯特（成都）生物制药股份有限公司 一种阳性脂质体dotap的制备方法
WO2019160383A1 (ko) 2018-02-19 2019-08-22 고려대학교 산학협력단 열충격단백질의 에피토프를 포함하는 백신 및 이의 용도
EP3539975A1 (en) 2018-03-15 2019-09-18 Fundació Privada Institut d'Investigació Oncològica de Vall-Hebron Micropeptides and uses thereof
EP3773505A4 (en) 2018-03-30 2021-12-22 Insmed Incorporated PROCESS FOR THE CONTINUOUS MANUFACTURING OF LIPOSOMAL MEDICINAL PRODUCTS
AU2019247490A1 (en) 2018-04-06 2020-10-22 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
US20210363547A1 (en) 2018-05-06 2021-11-25 Emendobio Inc. Differential knockout of an allele of a heterozygous elane gene
US12084423B2 (en) 2018-05-18 2024-09-10 Bioventures, Llc Piperlongumine analogues and uses thereof
US20210198330A1 (en) 2018-05-23 2021-07-01 The Broad Institute, Inc. Base editors and uses thereof
BR112020024863A2 (pt) 2018-06-05 2022-02-01 Lifeedit Inc Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso
EP3833761A1 (en) 2018-08-07 2021-06-16 The Broad Institute, Inc. Novel cas12b enzymes and systems
JP2021534101A (ja) 2018-08-09 2021-12-09 ヴェルソー セラピューティクス， インコーポレイテッド Ｃｃｒ２及びｃｓｆ１ｒを標的とするためのオリゴヌクレオチド組成物ならびにその使用
EP3835320A4 (en) 2018-08-10 2022-06-01 Eutilex Co., Ltd. BINDING OF CHEMERA ANTIGEN RECEPTOR TO HLA-DR, AND CAR-T CELL
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
CN118421617A (zh) 2018-08-24 2024-08-02 川斯勒佰尔公司 用于纯化信使rna的方法
US20210332367A1 (en) 2018-09-18 2021-10-28 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2020061161A1 (en) 2018-09-18 2020-03-26 Sangamo Therapeutics, Inc. Programmed cell death 1 (pd1) specific nucleases
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
CA3113472A1 (en) 2018-09-28 2020-04-02 President And Fellows Of Harvard College Cellular reprogramming to reverse aging and promote organ and tissue regeneration
GB201815820D0 (en) 2018-09-28 2018-11-14 Univ Wageningen Off-target activity inhibitors for guided endonucleases
US20220001030A1 (en) 2018-10-15 2022-01-06 Fondazione Telethon Genome editing methods and constructs
US20220389395A1 (en) 2018-10-29 2022-12-08 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
WO2020096927A1 (en) 2018-11-05 2020-05-14 Iovance Biotherapeutics, Inc. Expansion of tils utilizing akt pathway inhibitors
AU2019377422A1 (en) 2018-11-05 2021-05-27 Iovance Biotherapeutics, Inc. Treatment of NSCLC patients refractory for anti-PD-1 antibody
US20220090018A1 (en) 2018-11-05 2022-03-24 Iovance Biotherapeutics, Inc. Processes for production of tumor infiltrating lymphocytes and used of the same in immunotherapy
EP3877512A2 (en) 2018-11-05 2021-09-15 Iovance Biotherapeutics, Inc. Selection of improved tumor reactive t-cells
US20220282275A1 (en) 2018-11-15 2022-09-08 The Broad Institute, Inc. G-to-t base editors and uses thereof
US20220056444A1 (en) 2018-12-05 2022-02-24 Empirico Inc. Process to inhibit or eliminate eosinophilic diseases of the airway and related conditions
US11384344B2 (en) 2018-12-17 2022-07-12 The Broad Institute, Inc. CRISPR-associated transposase systems and methods of use thereof
JP2022514023A (ja) 2018-12-19 2022-02-09 アイオバンス バイオセラピューティクス，インコーポレイテッド 操作されたサイトカイン受容体対を使用して腫瘍浸潤リンパ球を拡大培養する方法及びその使用
CA3123617A1 (en) 2018-12-20 2020-06-25 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
BR112021012665A2 (pt) 2018-12-27 2021-11-03 Lifeedit Therapeutics Inc Polipeptídeos úteis para edição de genes e métodos de uso
WO2020150265A1 (en) 2019-01-15 2020-07-23 Empirico Inc. Prodrugs of alox-15 inhibitors and methods of using the same
US20220098621A1 (en) 2019-02-05 2022-03-31 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
WO2020163017A1 (en) 2019-02-06 2020-08-13 Klogenix Llc Dna binding proteins and uses thereof
WO2020163307A1 (en) 2019-02-06 2020-08-13 Emendobio Inc. New engineered high fidelity cas9
US20230053540A1 (en) 2019-02-19 2023-02-23 Massachusetts Institute Of Technology Treatment of liver injury
AU2020233284A1 (en) 2019-03-01 2021-09-16 Iovance Biotherapeutics, Inc. Expansion of tumor infiltrating lymphocytes from liquid tumors and therapeutic uses thereof
CA3131531A1 (en) 2019-03-03 2020-09-10 Prothena Biosciences Limited Antibodies recognizing tau
WO2020181195A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. T:a to a:t base editing through adenine excision
WO2020181180A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. A:t to c:g base editors and uses thereof
WO2020181202A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. A:t to t:a base editing through adenine deamination and oxidation
US20220170013A1 (en) 2019-03-06 2022-06-02 The Broad Institute, Inc. T:a to a:t base editing through adenosine methylation
WO2020181178A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. T:a to a:t base editing through thymine alkylation
MX2021010831A (es) 2019-03-08 2021-12-15 Obsidian Therapeutics Inc Composiciones de ligando del cumulo de diferenciación 40 (cd40l) y métodos para regulación ajustable.
GB201903499D0 (en) 2019-03-14 2019-05-01 Ucl Business Plc Minimal promoter
WO2020191102A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
MX2021012152A (es) 2019-04-02 2021-11-03 Sangamo Therapeutics Inc Metodos para el tratamiento de beta-talasemia.
CA3131759A1 (en) 2019-04-09 2020-10-15 Prashant MALI Long-lasting analgesia via targeted in vivo epigenetic repression
US11964001B2 (en) 2019-04-09 2024-04-23 Washington University Compositions for treating or preventing respiratory tract infections and method of use thereof
WO2020210751A1 (en) 2019-04-12 2020-10-15 The Broad Institute, Inc. System for genome editing
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP3958868A4 (en) 2019-04-22 2023-01-18 The Penn State Research Foundation METHODS AND COMPOSITIONS RELATING TO THE INHIBITION OF ALDEHYDE DEHYDROGENASES FOR THE TREATMENT OF CANCER
US20220213469A1 (en) 2019-04-30 2022-07-07 The Broad Institute, Inc. Methods and compositions for barcoding nucleic acid libraries and cell populations
WO2020232029A1 (en) 2019-05-13 2020-11-19 Iovance Biotherapeutics, Inc. Methods and compositions for selecting tumor infiltrating lymphocytes and uses of the same in immunotherapy
US20220249701A1 (en) 2019-05-14 2022-08-11 The Broad Institute, Inc. Compositions and methods for targeting multinucleated cells
AU2020279101A1 (en) 2019-05-17 2021-11-18 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
US20220249697A1 (en) 2019-05-20 2022-08-11 The Broad Institute, Inc. Aav delivery of nucleobase editors
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
WO2020243371A1 (en) 2019-05-28 2020-12-03 Massachusetts Institute Of Technology Methods and compositions for modulating immune responses
US11447790B2 (en) 2019-05-29 2022-09-20 Altria Client Services Llc Compositions and methods for producing tobacco plants and products having reduced or eliminated suckers
US20210317192A9 (en) 2019-05-29 2021-10-14 Massachusetts Institute Of Technology Hiv-1 specific immunogen compositions and methods of use
TW202113074A (zh) 2019-06-07 2021-04-01 美商斯奎柏治療公司 工程化之ｃａｓｘ系統
GB201909597D0 (en) 2019-07-03 2019-08-14 Univ Wageningen Crispr type v-u1 system from mycobacterium mucogenicum and uses thereof
CN114222815A (zh) 2019-07-23 2022-03-22 记忆疗法公司 Suv39h1缺陷的免疫细胞
WO2021025750A1 (en) 2019-08-08 2021-02-11 The Broad Institute, Inc. Base editors with diversified targeting scope
BR112022002695A2 (pt) 2019-08-12 2022-08-23 Lifeedit Therapeutics Inc Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
WO2021030666A1 (en) 2019-08-15 2021-02-18 The Broad Institute, Inc. Base editing by transglycosylation
JP2022544688A (ja) 2019-08-16 2022-10-20 マサチューセッツ インスティテュート オブ テクノロジー Ｃｒｉｓｐｒ／ｃａｓ１３を使用する標的化トランススプライシング
KR102099342B1 (ko) 2019-09-03 2020-04-10 주식회사 제노포커스 Crm197 단백질 발현 방법
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
US11827893B2 (en) 2019-10-10 2023-11-28 Altria Client Services Llc Pale yellow locus and its applications in tobacco
AU2020376048A1 (en) 2019-11-01 2022-06-02 Sangamo Therapeutics, Inc. Compositions and methods for genome engineering
CN114728018B (zh) 2019-11-01 2024-07-19 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
EP4055167A2 (en) 2019-11-08 2022-09-14 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides targeting bromodomain containing protein 4 (brd4) for immunotherapy
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
WO2021108717A2 (en) 2019-11-26 2021-06-03 The Broad Institute, Inc Systems and methods for evaluating cas9-independent off-target editing of nucleic acids
CA3161104A1 (en) 2019-12-11 2021-06-17 Cecile Chartier-Courtaud Processes for the production of tumor infiltrating lymphocytes (tils) and methods of using the same
WO2021119226A1 (en) 2019-12-13 2021-06-17 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
AU2020409124A1 (en) 2019-12-20 2022-06-30 Novarock Biotherapeutics, Ltd. Anti-interleukin-23 p19 antibodies and methods of use thereof
EP4085133A1 (en) 2019-12-30 2022-11-09 Lifeedit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
EP4085136A1 (en) 2019-12-31 2022-11-09 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides with improved systemic delivery
US20230056856A1 (en) 2020-01-08 2023-02-23 Obsidian Therapeutics, Inc. Compositions and methods for tunable regulation of transcription
GB202000934D0 (en) 2020-01-22 2020-03-04 Ucl Business Ltd Engineered immune cells
WO2021155065A1 (en) 2020-01-28 2021-08-05 The Broad Institute, Inc. Base editors, compositions, and methods for modifying the mitochondrial genome
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
US20230235309A1 (en) 2020-02-05 2023-07-27 The Broad Institute, Inc. Adenine base editors and uses thereof
US20230108687A1 (en) 2020-02-05 2023-04-06 The Broad Institute, Inc. Gene editing methods for treating spinal muscular atrophy
WO2021163191A1 (en) 2020-02-10 2021-08-19 Board Of Regents, The University Of Texas System Methods for rapid cloning and expression of hla class i cells
EP4103687A4 (en) 2020-02-11 2024-03-20 Tenza, Inc. BIOTHERAPEUTICS FOR THE DETECTION, DIAGNOSIS AND TREATMENT OF DISEASES ASSOCIATED WITH MUCOUS BLEEDING
TW202140069A (zh) 2020-02-14 2021-11-01 美商默沙東藥廠 Hpv疫苗
US11752199B1 (en) 2020-02-25 2023-09-12 Mallinckrodt Ard Ip Unlimited Company Methods of modulating pericytes
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
EP3885440A1 (en) 2020-03-26 2021-09-29 Splicebio, S.L. Split inteins and their uses
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
TW202208626A (zh) 2020-04-24 2022-03-01 美商生命編輯公司 Rna引導核酸酶及其活性片段與變體，以及使用方法
JP2023523993A (ja) 2020-04-27 2023-06-08 アルナイラム ファーマシューティカルズ， インコーポレイテッド アポリポタンパク質Ｅ（ＡｐｏＥ）ｉＲＮＡ剤組成物およびその使用方法
WO2021222318A1 (en) 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
EP4146794A1 (en) 2020-05-04 2023-03-15 Iovance Biotherapeutics, Inc. Processes for production of tumor infiltrating lymphocytes and uses of the same in immunotherapy
US20230212613A1 (en) 2020-05-06 2023-07-06 Cellectis S.A. Methods for targeted insertion of exogenous sequences in cellular genomes
CN115715203A (zh) 2020-05-06 2023-02-24 塞勒克提斯公司 对细胞进行基因修饰以递送治疗性蛋白质的方法
CN111393595A (zh) * 2020-05-06 2020-07-10 东莞市竤穗实业投资有限公司 一种具有温敏特性的生物基聚碳化二亚胺交联剂及其制备方法
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
CA3173882A1 (en) 2020-05-11 2021-11-18 Alexandra Briner CRAWLEY Rna-guided nucleic acid binding proteins and active fragments and variants thereof and methods of use
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231673A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
CA3178760A1 (en) 2020-05-19 2021-11-25 Robin Barbour Multi-epitope vaccine for the treatment of alzheimer's disease
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
JP2023533458A (ja) 2020-06-24 2023-08-03 プロシーナ バイオサイエンシーズ リミテッド ソルチリンを認識する抗体
CN116113697A (zh) 2020-07-10 2023-05-12 国家健康与医学研究院 用于治疗癫痫的方法和组合物
WO2022015969A1 (en) 2020-07-15 2022-01-20 LifeEDIT Therapeutics, Inc. Uracil stabilizing proteins and active fragments and variants thereof and methods of use
AU2021316727A1 (en) 2020-07-30 2023-03-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Immune cells defective for SOCS1
WO2022034374A2 (en) 2020-08-11 2022-02-17 University Of Oslo Improved gene editing
EP4204545A2 (en) 2020-08-25 2023-07-05 Kite Pharma, Inc. T cells with improved functionality
JP2023540526A (ja) 2020-09-04 2023-09-25 ノヴァロック バイオセラピューティクス， リミテッド ネクチン－４抗体およびそれの使用
AU2021338707A1 (en) 2020-09-10 2023-04-06 Monsanto Technology Llc Increasing gene editing and site-directed integration events utilizing meiotic and germline promoters
CA3173886A1 (en) 2020-09-11 2022-03-17 Tyson D. BOWEN Dna modifying enzymes and active fragments and variants thereof and methods of use
WO2022060927A1 (en) 2020-09-17 2022-03-24 Northwestern University Engineered mammalian genetic circuits and methods of using the same
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
EP3973978A1 (en) 2020-09-25 2022-03-30 Mallinckrodt Ard IP Unlimited Company Methods of modulating corticosteroid response
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
WO2022076606A1 (en) 2020-10-06 2022-04-14 Iovance Biotherapeutics, Inc. Treatment of nsclc patients with tumor infiltrating lymphocyte therapies
EP4225330A1 (en) 2020-10-06 2023-08-16 Iovance Biotherapeutics, Inc. Treatment of nsclc patients with tumor infiltrating lymphocyte therapies
EP4229074A1 (en) 2020-10-16 2023-08-23 Stichting Het Nederlands Kanker Instituut- Antoni van Leeuwenhoek Ziekenhuis Anti-tumor immunity induces the presentation of aberrant peptides
AU2021365822A1 (en) 2020-10-21 2023-06-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
EP4251170A1 (en) 2020-11-25 2023-10-04 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
AU2021393417A1 (en) 2020-12-01 2023-06-29 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
JP2024500403A (ja) 2020-12-17 2024-01-09 アイオバンス バイオセラピューティクス，インコーポレイテッド 腫瘍浸潤リンパ球によるがんの治療
EP4262811A1 (en) 2020-12-17 2023-10-25 Iovance Biotherapeutics, Inc. Treatment with tumor infiltrating lymphocyte therapies in combination with ctla-4 and pd-1 inhibitors
WO2022150790A2 (en) 2021-01-11 2022-07-14 The Broad Institute, Inc. Prime editor variants, constructs, and methods for enhancing prime editing efficiency and precision
EP4284925A1 (en) 2021-01-26 2023-12-06 California Institute of Technology Allosteric conditional guide rnas for cell-selective regulation of crispr/cas
EP4284923A1 (en) 2021-01-29 2023-12-06 Wageningen Universiteit Crispr-associated base-editing of the complementary strand
US20220243216A1 (en) 2021-02-03 2022-08-04 Altria Client Services Llc Increasing trichome density and improving transport of metabolites in plant trichomes
TW202245835A (zh) 2021-02-04 2022-12-01 美商默沙東有限責任公司 用於肺炎球菌結合物疫苗之奈米乳化液佐劑組合物
IL304880A (en) 2021-02-12 2023-10-01 Alnylam Pharmaceuticals Inc Superoxide dismutase 1 (SOD1) IRNA compositions and methods of using them to treat or prevent superoxide dismutase 1- (SOD1-) associated neurodegenerative diseases
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
EP4304637A1 (en) 2021-03-12 2024-01-17 Synthego Corporation Genetically modified cells expressing antigen-containing fusion proteins and uses thereof
AR125191A1 (es) 2021-03-22 2023-06-21 Lifeedit Therapeutics Inc Enzimas modificadoras del adn y sus fragmentos activos y variantes y métodos de uso
MX2023011466A (es) 2021-03-29 2024-02-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de ácido ribonucleico de interferencia (arni) de huntingtina (htt) y métodos de uso de estas.
TW202308669A (zh) 2021-04-19 2023-03-01 美商艾歐凡斯生物治療公司 嵌合共刺激性受體、趨化激素受體及彼等於細胞免疫治療之用途
CA3214540A1 (en) 2021-04-22 2022-10-27 Pasi A. Janne Compositions and methods for treating cancer
JP2024519293A (ja) 2021-04-29 2024-05-10 アルナイラム ファーマシューティカルズ， インコーポレイテッド シグナル伝達兼転写活性化因子６（ＳＴＡＴ６）ｉＲＮＡ組成物およびその使用方法
JP2024522068A (ja) 2021-05-18 2024-06-11 アルナイラム ファーマシューティカルズ， インコーポレイテッド ナトリウム－グルコース共輸送体２（ｓｇｌｔ２）ｉｒｎａ組成物およびその使用方法
EP4341405A1 (en) 2021-05-20 2024-03-27 Korro Bio, Inc. Methods and compositions for adar-mediated editing
AU2022277649A1 (en) 2021-05-21 2023-11-30 Cellectis S.A. Enhancing efficacy of t-cell-mediated immunotherapy by modulating cancer-associated fibroblasts in solid tumors
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
BR112023025224A2 (pt) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos
WO2022261394A1 (en) 2021-06-11 2022-12-15 LifeEDIT Therapeutics, Inc. Rna polymerase iii promoters and methods of use
WO2022261509A1 (en) 2021-06-11 2022-12-15 The Broad Institute, Inc. Improved cytosine to guanine base editors
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
WO2023288287A2 (en) 2021-07-15 2023-01-19 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs and methods of use for cell rejuvenation and for treatment
WO2023288288A1 (en) 2021-07-15 2023-01-19 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs with on/off mechanism for controlled expression and methods of use
US20230042860A1 (en) 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Polycistronic expression vectors
GB202110644D0 (en) 2021-07-23 2021-09-08 Sisaf Ltd Improved nucleic acid vector particles
IL309905A (en) 2021-07-23 2024-03-01 Alnylam Pharmaceuticals Inc IRNA compositions in β-catenin (CTNNB1) and methods of using them
GB202110646D0 (en) 2021-07-23 2021-09-08 Sisaf Ltd Nucleic acid vector compositions
EP4377446A1 (en) 2021-07-28 2024-06-05 Iovance Biotherapeutics, Inc. Treatment of cancer patients with tumor infiltrating lymphocyte therapies in combination with kras inhibitors
WO2023015264A1 (en) 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
US20240301430A1 (en) 2021-08-04 2024-09-12 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides
EP4381057A2 (en) 2021-08-06 2024-06-12 The Broad Institute Inc. Improved prime editors and methods of use
US20230118665A1 (en) 2021-08-19 2023-04-20 Merck Sharp & Dohme Llc Novel thermostable lipid nanoparticle and methods of use thereof
AU2022357132A1 (en) 2021-10-01 2024-04-18 Koninklijke Nederlandse Akademie Van Wetenschappen Mammalian cardiac regeneration
EP4419683A1 (en) 2021-10-22 2024-08-28 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
KR20240090727A (ko) 2021-10-22 2024-06-21 세일 바이오메디슨스, 인크. Mrna 백신 조성물
WO2023076898A1 (en) 2021-10-25 2023-05-04 The Broad Institute, Inc. Methods and compositions for editing a genome with prime editing and a recombinase
EP4423755A2 (en) 2021-10-27 2024-09-04 Iovance Biotherapeutics, Inc. Systems and methods for coordinating manufacturing of cells for patient-specific immunotherapy
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
IL312452A (en) 2021-11-01 2024-06-01 Tome Biosciences Inc A transformant has a single structure for the simultaneous transfer of a gene editing mechanism and a nucleic acid cargo
CN114015674B (zh) 2021-11-02 2024-08-30 辉大(上海)生物科技有限公司 一种CRISPR-Cas12i系统
WO2023080788A1 (en) 2021-11-05 2023-05-11 Stichting Het Nederlands Kanker Instituut-Antoni van Leeuwenhoek Ziekenhuis Tryptophan depletion induces production and presentation of tryptophan to phenylalanine substitutions
EP4430167A1 (en) 2021-11-10 2024-09-18 Iovance Biotherapeutics, Inc. Methods of expansion treatment utilizing cd8 tumor infiltrating lymphocytes
CA3238764A1 (en) 2021-11-23 2023-06-01 Siddharth Patel A bacteria-derived lipid composition and use thereof
WO2023096494A1 (en) 2021-11-26 2023-06-01 Synvolux Ip B.V. Conserved coronavirus t cell epitopes
CA3241014A1 (en) 2021-12-20 2023-06-29 Munir MOSAHEB Mrna therapeutic compositions
WO2023122762A1 (en) 2021-12-22 2023-06-29 Camp4 Therapeutics Corporation Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
AU2022420615A1 (en) 2021-12-22 2024-07-04 Tome Biosciences, Inc. Co-delivery of a gene editor construct and a donor template
WO2023126458A1 (en) 2021-12-28 2023-07-06 Mnemo Therapeutics Immune cells with inactivated suv39h1 and modified tcr
WO2023139557A1 (en) 2022-01-24 2023-07-27 LifeEDIT Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
AU2023216314A1 (en) 2022-02-02 2024-08-22 Inscripta, Inc. Nucleic acid-guided nickase fusion proteins
AU2023248451A1 (en) 2022-04-04 2024-10-17 President And Fellows Of Harvard College Cas9 variants having non-canonical pam specificities and uses thereof
WO2023196818A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California Genetic complementation compositions and methods
WO2023196851A1 (en) 2022-04-06 2023-10-12 President And Fellows Of Harvard College Reversing aging of the central nervous system
WO2023201369A1 (en) 2022-04-15 2023-10-19 Iovance Biotherapeutics, Inc. Til expansion processes using specific cytokine combinations and/or akti treatment
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023212715A1 (en) 2022-04-28 2023-11-02 The Broad Institute, Inc. Aav vectors encoding base editors and uses thereof
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
WO2023220603A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2023220457A1 (en) 2022-05-13 2023-11-16 Northwestern University Receptor engagement-mediated enhancement of biologics delivery
WO2023220392A1 (en) 2022-05-13 2023-11-16 Northwestern University Synthetic modular extracellular sensors that employ natural receptor ligand-binding domains
WO2023220459A1 (en) 2022-05-13 2023-11-16 Northwestern University Active loading of cargo entity into lipid bilayer particles using dimerization domains
EP4279085A1 (en) 2022-05-20 2023-11-22 Mnemo Therapeutics Compositions and methods for treating a refractory or relapsed cancer or a chronic infectious disease
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2023230433A1 (en) 2022-05-23 2023-11-30 Altria Client Services Llc Methods and compositions for regulating alkaloids in tobacco field
WO2023230587A2 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2023230613A1 (en) 2022-05-27 2023-11-30 The Broad Institute, Inc. Improved mitochondrial base editors and methods for editing mitochondrial dna
TW202411426A (zh) 2022-06-02 2024-03-16 美商斯奎柏治療公司 經工程化的２類ｖ型ｃｒｉｓｐｒ系統
US20230392188A1 (en) 2022-06-02 2023-12-07 Perkinelmer Health Sciences, Inc. Electrophoresis-mediated characterization of dna content of adeno-associated virus capsids
EP4314267A1 (en) 2022-06-07 2024-02-07 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
TW202413643A (zh) 2022-06-07 2024-04-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法
WO2023240137A1 (en) 2022-06-08 2023-12-14 The Board Institute, Inc. Evolved cas14a1 variants, compositions, and methods of making and using same in genome editing
WO2023240277A2 (en) 2022-06-10 2023-12-14 Camp4 Therapeutics Corporation Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas
CN115557851B (zh) * 2022-06-27 2023-04-18 上海云沂生物医药科技有限公司 一种氨基脂质、其合成方法、颗粒及用途
WO2024003334A1 (en) 2022-06-30 2024-01-04 Cellectis S.A. Enhancing safety of t-cell-mediated immunotherapy
WO2024015383A1 (en) 2022-07-12 2024-01-18 Northwestern University Engineered hypoxia biosensors and methods of using the same
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
US20240229054A9 (en) 2022-08-05 2024-07-11 Altria Client Services Llc Methods and compositions for regulating alkaloids in tobacco
WO2024033901A1 (en) 2022-08-12 2024-02-15 LifeEDIT Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2024040083A1 (en) 2022-08-16 2024-02-22 The Broad Institute, Inc. Evolved cytosine deaminases and methods of editing dna using same
TW202424186A (zh) 2022-08-25 2024-06-16 美商生命編輯治療學公司 Rna引導核酸酶中介基因編輯用之具鎖核酸引導 rna之化學修飾
WO2024047587A1 (en) 2022-08-31 2024-03-07 Regel Therapeutics, Inc. Cas-phi compositions and methods of use
TW202424193A (zh) 2022-09-15 2024-06-16 美商艾拉倫製藥股份有限公司 第１３型１７β－羥基類固醇去氫酶（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物及其使用方法
WO2024094775A1 (en) 2022-11-03 2024-05-10 Cellectis S.A. Enhancing efficacy and safety of t-cell-mediated immunotherapy
WO2024095245A2 (en) 2022-11-04 2024-05-10 LifeEDIT Therapeutics, Inc. Evolved adenine deaminases and rna-guided nuclease fusion proteins with internal insertion sites and methods of use
US20240182561A1 (en) 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024098024A1 (en) 2022-11-04 2024-05-10 Iovance Biotherapeutics, Inc. Expansion of tumor infiltrating lymphocytes from liquid tumors and therapeutic uses thereof
WO2024102434A1 (en) 2022-11-10 2024-05-16 Senda Biosciences, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2024119145A1 (en) 2022-12-01 2024-06-06 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2024127369A1 (en) 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target foxp3 gene and methods of use
WO2024127370A1 (en) 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target trac gene and methods of use
GB202219855D0 (en) 2022-12-30 2023-02-15 Univ Oxford Innovation Ltd Affinity capture reagents for mass spectrometry
WO2024155597A1 (en) 2023-01-17 2024-07-25 Inscripta, Inc. Methods and compositions of co-expression of t7rna polymerase and inhibitory rna aptamers
GB202300915D0 (en) 2023-01-20 2023-03-08 Sisaf Ltd mRNA COMPOSITIONS
WO2024159172A1 (en) 2023-01-27 2024-08-02 Senda Biosciences, Inc. A modified lipid composition and uses thereof
WO2024163862A2 (en) 2023-02-03 2024-08-08 The Broad Institute, Inc. Gene editing methods, systems, and compositions for treating spinal muscular atrophy
WO2024206565A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Repressor fusion protein systems
WO2024206676A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa
WO2024206555A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3931397A (en) * 1971-11-05 1976-01-06 Beecham Group Limited Biologically active material
IT1196363B (it) * 1984-12-10 1988-11-16 Bracco Ind Chimica Spa Derivati dell'acido 5-alcossi-2,4,6-triiodo- o -tribromo-isoftalico,metodo per la loro preparazione e relativi mezzi di contrasto radiologici
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
EP0187702B1 (en) * 1985-01-07 1990-03-14 Syntex (U.S.A.) Inc. N-(omega,omega-1-dialkoxy)- and n-(omega,omega-1-dialkenoxy)-alk-1-yl-n,n,n-trisubstituted ammonium surfactants, their preparation and pharmaceutical formulations containing them
JPS63102682A (ja) * 1986-10-20 1988-05-07 Medeisa Shinyaku Kk 真核細胞内へのｄｎａ導入法
US4840738A (en) * 1988-02-25 1989-06-20 The Procter & Gamble Company Stable biodegradable fabric softening compositions containing 2-hydroxypropyl monoester quaternized ammonium salts
CA2001401A1 (en) * 1988-10-25 1990-04-25 Claude Piantadosi Quaternary amine containing ether or ester lipid derivatives and therapeutic compositions
JP2805092B2 (ja) * 1989-09-14 1998-09-30 ソニー株式会社 ディスクドライブ装置
JP2604268B2 (ja) * 1990-04-09 1997-04-30 富士写真フイルム株式会社 ペプチド誘導体両親媒性化合物、その中間体、ペプチド誘導体両親媒性化合物を用いたリポソームおよび薄膜
DE4013632A1 (de) * 1990-04-27 1991-10-31 Max Planck Gesellschaft Liposomen mit positiver ueberschussladung
AU7979491A (en) * 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes

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EP0523189A4 (es) 1995-04-26
DK0523189T3 (da) 1999-12-13
DE69131347T2 (de) 2000-03-30
GR3030974T3 (en) 1999-12-31
CA2079814A1 (en) 1991-10-20
CA2079814C (en) 2005-09-27
JP2538474B2 (ja) 1996-09-25
AU7854791A (en) 1991-11-11
US5264618A (en) 1993-11-23
ATE181319T1 (de) 1999-07-15
WO1991016024A1 (en) 1991-10-31
DE69131347D1 (de) 1999-07-22
EP0523189A1 (en) 1993-01-20
JPH05508626A (ja) 1993-12-02
US5459127A (en) 1995-10-17
EP0523189B1 (en) 1999-06-16

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