MX9303965A - Nucleosidos carbociclicos que contienen anillos biciclicos, oligonucleotidos a partir de los mismos, procedimiento para su preparacion, su uso y sus intermediarios. - Google Patents

Nucleosidos carbociclicos que contienen anillos biciclicos, oligonucleotidos a partir de los mismos, procedimiento para su preparacion, su uso y sus intermediarios.

Info

Publication number
MX9303965A
MX9303965A MX9303965A MX9303965A MX9303965A MX 9303965 A MX9303965 A MX 9303965A MX 9303965 A MX9303965 A MX 9303965A MX 9303965 A MX9303965 A MX 9303965A MX 9303965 A MX9303965 A MX 9303965A
Authority
MX
Mexico
Prior art keywords
preparation
oligonucleotides
carbociclic
intermediaries
procedure
Prior art date
Application number
MX9303965A
Other languages
English (en)
Spanish (es)
Inventor
Karl-Heinz Altmann
Rene Imwinkelried
Albert Eschenmoser
Original Assignee
Ciba Geigy Ag
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 Ciba Geigy Ag filed Critical Ciba Geigy Ag
Publication of MX9303965A publication Critical patent/MX9303965A/es

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/46Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/47One nitrogen atom and one oxygen or sulfur atom, e.g. cytosine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C215/00Compounds containing amino and hydroxy groups bound to the same carbon skeleton
    • C07C215/42Compounds containing amino and hydroxy groups bound to the same carbon skeleton having amino groups or hydroxy groups bound to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton
    • C07C215/44Compounds containing amino and hydroxy groups bound to the same carbon skeleton having amino groups or hydroxy groups bound to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton bound to carbon atoms of the same ring or condensed ring system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C275/00Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups
    • C07C275/46Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups containing any of the groups, X being a hetero atom, Y being any atom, e.g. acylureas
    • C07C275/48Y being a hydrogen or a carbon atom
    • C07C275/50Y being a hydrogen or an acyclic carbon atom
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/46Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/48Two nitrogen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/46Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/50Three nitrogen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D239/00Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/46Two or more oxygen, sulphur or nitrogen atoms
    • C07D239/52Two oxygen atoms
    • C07D239/54Two oxygen atoms as doubly bound oxygen atoms or as unsubstituted hydroxy radicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D473/00Heterocyclic compounds containing purine ring systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F7/00Compounds containing elements of Groups 4 or 14 of the Periodic Table
    • C07F7/02Silicon compounds
    • C07F7/08Compounds having one or more C—Si linkages
    • C07F7/18Compounds having one or more C—Si linkages as well as one or more C—O—Si linkages
    • C07F7/1804Compounds having Si-O-C linkages
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/645Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having two nitrogen atoms as the only ring hetero atoms
    • C07F9/6509Six-membered rings
    • C07F9/6512Six-membered rings having the nitrogen atoms in positions 1 and 3
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • C07F9/65616Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02PCLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00Technologies relating to chemical industry
    • Y02P20/50Improvements relating to the production of bulk chemicals
    • Y02P20/55Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
MX9303965A 1992-07-01 1993-06-30 Nucleosidos carbociclicos que contienen anillos biciclicos, oligonucleotidos a partir de los mismos, procedimiento para su preparacion, su uso y sus intermediarios. MX9303965A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CH207592 1992-07-01

Publications (1)

Publication Number Publication Date
MX9303965A true MX9303965A (es) 1994-04-29

Family

ID=4225321

Family Applications (1)

Application Number Title Priority Date Filing Date
MX9303965A MX9303965A (es) 1992-07-01 1993-06-30 Nucleosidos carbociclicos que contienen anillos biciclicos, oligonucleotidos a partir de los mismos, procedimiento para su preparacion, su uso y sus intermediarios.

Country Status (5)

Country Link
US (3) US5461152A (enExample)
EP (1) EP0577558A2 (enExample)
JP (1) JPH06100562A (enExample)
CA (1) CA2099712A1 (enExample)
MX (1) MX9303965A (enExample)

Families Citing this family (773)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US8153602B1 (en) 1991-11-19 2012-04-10 Isis Pharmaceuticals, Inc. Composition and methods for the pulmonary delivery of nucleic acids
DE69433036T2 (de) 1993-09-03 2004-05-27 Isis Pharmaceuticals, Inc., Carlsbad Aminoderivatisierte nukleoside und oligonukleoside
DE69406943T2 (de) * 1993-09-24 1998-05-07 Us Health Konformationell festgelegte nukleosidanaloge
JP3769737B2 (ja) * 1994-03-30 2006-04-26 味の素株式会社 シクロプロパン誘導体及びその製造法
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US20040147022A1 (en) * 1996-06-06 2004-07-29 Baker Brenda F. 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
US20030044941A1 (en) 1996-06-06 2003-03-06 Crooke Stanley T. Human RNase III and compositions and uses thereof
US20040203024A1 (en) * 1996-06-06 2004-10-14 Baker Brenda F. Modified oligonucleotides for use in RNA interference
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US20050053976A1 (en) * 1996-06-06 2005-03-10 Baker Brenda F. Chimeric oligomeric compounds and their use in gene modulation
WO2005121371A2 (en) * 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
US7812149B2 (en) * 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US20040171031A1 (en) * 1996-06-06 2004-09-02 Baker Brenda F. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
AU6766496A (en) * 1996-08-07 1998-02-25 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Conformationally locked nucleoside analogs as antiherpetic agents
US6639062B2 (en) 1997-02-14 2003-10-28 Isis Pharmaceuticals, Inc. Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom
US6716625B1 (en) 1997-04-16 2004-04-06 Claude Selitrennikoff Histidine kinases of Aspergillus and other fungal species, related compositions, and methods of use
CA2294988C (en) 1997-07-01 2015-11-24 Isis Pharmaceuticals Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
IL133800A0 (en) 1997-07-03 2001-04-30 Du Pont Pharm Co Imidazopyrimidines and imidazopyridines for the treatment of neurological disorders
US7321828B2 (en) * 1998-04-13 2008-01-22 Isis Pharmaceuticals, Inc. System of components for preparing oligonucleotides
US20040186071A1 (en) * 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
CA2329252A1 (en) 1998-05-21 1999-11-25 Isis Pharmaceuticals Inc. Compositions and methods for topical delivery of oligonucleotides
CA2329130A1 (en) * 1998-05-21 1999-11-25 Isis Pharmaceuticals Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
US6335439B1 (en) 1998-06-11 2002-01-01 Isis Pharmaceuticals, Inc. Method of preparing phosphoramidites
US6124463A (en) * 1998-07-02 2000-09-26 Dupont Pharmaceuticals Benzimidazoles as corticotropin release factor antagonists
US6365589B1 (en) 1998-07-02 2002-04-02 Bristol-Myers Squibb Pharma Company Imidazo-pyridines, -pyridazines, and -triazines as corticotropin releasing factor antagonists
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US6225293B1 (en) 1998-09-02 2001-05-01 Isis Pharmaceuticals, Inc. Methods and compounds for tracking the biodistribution of macromolecule-carrier combinations
US6077709A (en) 1998-09-29 2000-06-20 Isis Pharmaceuticals Inc. Antisense modulation of Survivin expression
US6300320B1 (en) 1999-01-05 2001-10-09 Isis Pharmaceuticals, Inc. Modulation of c-jun using inhibitors of protein kinase C
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US6252843B1 (en) 1999-04-09 2001-06-26 Hewlett Packard Company Automatic clamping of compact discs
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
US6147200A (en) * 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US7668658B2 (en) * 1999-10-13 2010-02-23 Sequenom, Inc. Methods for generating databases and databases for identifying polymorphic genetic markers
EP1252160B1 (en) * 2000-01-14 2006-08-16 The Government of the United States of America, as represented by the Secretary of the Department of Health and Human Services Methanocarba cycloalkyl nucleoside analogues
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
US20030176385A1 (en) * 2000-02-15 2003-09-18 Jingfang Ju Antisense modulation of protein expression
JP2003530841A (ja) * 2000-04-13 2003-10-21 トーマス エヌ. ワイト, 治療的化合物および方法
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
US20060166227A1 (en) * 2000-06-20 2006-07-27 Stephen Kingsmore Protein expression profiling
US6323009B1 (en) * 2000-06-28 2001-11-27 Molecular Staging, Inc. Multiply-primed amplification of nucleic acid sequences
US6958214B2 (en) 2000-07-10 2005-10-25 Sequenom, Inc. Polymorphic kinase anchor proteins and nucleic acids encoding the same
US8568766B2 (en) * 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
AU2001296846B2 (en) 2000-10-12 2007-07-05 University Of Rochester Compositions that inhibit proliferation of cancer cells
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US6573051B2 (en) * 2001-03-09 2003-06-03 Molecular Staging, Inc. Open circle probes with intramolecular stem structures
ES2328796T3 (es) 2001-03-14 2009-11-18 Myriad Genetics, Inc. Interaccion tsg101-gag y uso de la misma.
KR100788092B1 (ko) 2001-06-20 2007-12-21 제넨테크, 인크. 종양의 진단 및 치료를 위한 방법 및 이를 위한 조성물
US7803915B2 (en) * 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
US20050107595A1 (en) * 2001-06-20 2005-05-19 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
WO2003000707A2 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense 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
EP2143438B1 (en) 2001-09-18 2011-07-13 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumors
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
NZ577565A (en) 2001-10-09 2010-10-29 Isis Pharmaceuticals Inc Antisense modulation of insulin-like growth factor binding protein 5 expressions
WO2003054143A2 (en) * 2001-10-25 2003-07-03 Neurogenetics, Inc. Genes and polymorphisms on chromosome 10 associated with alzheimer's disease and other neurodegenerative diseases
US20030170678A1 (en) * 2001-10-25 2003-09-11 Neurogenetics, Inc. Genetic markers for Alzheimer's disease and methods using the same
US20030224380A1 (en) * 2001-10-25 2003-12-04 The General Hospital Corporation Genes and polymorphisms on chromosome 10 associated with Alzheimer's disease and other neurodegenerative diseases
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
EP2067472A1 (en) 2002-01-02 2009-06-10 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
US7553619B2 (en) * 2002-02-08 2009-06-30 Qiagen Gmbh Detection method using dissociated rolling circle amplification
US7169916B2 (en) * 2002-04-01 2007-01-30 Isis Pharmaceuticals, Inc. Chloral-free DCA in oligonucleotide synthesis
AU2003230874A1 (en) 2002-04-16 2003-11-03 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
WO2003093296A2 (en) * 2002-05-03 2003-11-13 Sequenom, Inc. Kinase anchor protein muteins, peptides thereof, and related methods
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
US20040092470A1 (en) * 2002-06-18 2004-05-13 Leonard Sherry A. Dry powder oligonucleotide formualtion, preparation and its uses
WO2004013160A2 (en) 2002-08-05 2004-02-12 University Of Rochester Protein transducing domain/deaminase chimeric proteins, related compounds, and uses thereof
BR0314236A (pt) 2002-09-13 2005-08-09 Replicor Inc Formulação de oligonucleotìdeo, composição farmacêutica, kit, composto antiviral, preparação de oligonucleotìdeo e métodos para seleção de um oligonucleotìdeo antiviral para uso como um agente antiviral, para profilaxia ou tratamento de uma infecção viral em um paciente, para tratamento profilático de câncer causado por oncovìrus, para identificação de um composto que altera a ligação de um oligonucleotìdeo a pelo menos um componente viral, para purificação da ligação de oligonucleotìdeos a pelo menos um componente viral e para enriquecimento de oligonucleotìdeos a partir de um agrupamento de oligonucleotìdeos
EP2322535A3 (en) 2002-09-20 2011-09-28 Yale University Riboswitches, methods for their use, and compositions for use with riboswitches
EP1549767A4 (en) 2002-09-26 2006-06-07 Amgen Inc MODULATION OF EXPRESSION OF FORKHEAD BOX O1A
AU2003304203A1 (en) * 2002-10-29 2005-01-04 Pharmacia Corporation Differentially expressed genes involved in cancer, the polypeptides encoded thereby, and methods of using the same
US9150606B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
AU2003290598A1 (en) 2002-11-05 2004-06-03 Isis Pharmaceuticals, Inc. Modified oligonucleotides for use in rna interference
WO2004044138A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
PT2336318E (pt) 2002-11-13 2013-06-12 Genzyme Corp Modulação anti-sentido da expressão de apolipoproteína b
EP2336318B1 (en) 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
EP1572971B1 (en) * 2002-11-15 2009-09-30 Morphotek Inc. Methods of generating high-production of antibodies from hybridomas created by in vitro immunization
WO2004045520A2 (en) 2002-11-15 2004-06-03 Musc Foundation For Research Development Complement receptor 2 targeted complement modulators
US7557092B2 (en) 2002-11-21 2009-07-07 University Of Utah Research Foundation Purinergic modulation of smell
US7144999B2 (en) 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
US20040115643A1 (en) * 2002-12-12 2004-06-17 Lizardi Paul M. Thermodynamic equilibrium extension of primers
US20040121338A1 (en) * 2002-12-19 2004-06-24 Alsmadi Osama A. Real-time detection of rolling circle amplification products
JP4886298B2 (ja) 2002-12-20 2012-02-29 キアゲン ゲゼルシャフト ミット ベシュレンクテル ハフツング 核酸増幅
US9487823B2 (en) 2002-12-20 2016-11-08 Qiagen Gmbh Nucleic acid amplification
US6977153B2 (en) 2002-12-31 2005-12-20 Qiagen Gmbh Rolling circle amplification of RNA
US7468356B2 (en) 2003-02-11 2008-12-23 Antisense Therapeutics Ltd. Modulation of insulin like growth factor I receptor expression
US7002006B2 (en) 2003-02-12 2006-02-21 Isis Pharmaceuticals, Inc. Protection of nucleosides
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US20070141570A1 (en) * 2003-03-07 2007-06-21 Sequenom, Inc. Association of polymorphic kinase anchor proteins with cardiac phenotypes and related methods
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
US8043834B2 (en) 2003-03-31 2011-10-25 Qiagen Gmbh Universal reagents for rolling circle amplification and methods of use
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
CA2540692C (en) * 2003-06-02 2013-05-28 Isis Pharmaceuticals, Inc. Oligonucleotide synthesis with alternative solvents
US7541344B2 (en) 2003-06-03 2009-06-02 Eli Lilly And Company Modulation of survivin expression
US7786290B2 (en) 2003-06-13 2010-08-31 Alnylam Pharmaceuticals, Inc. Double-stranded ribonucleic acid with increased effectiveness in an organism
EP1636342A4 (en) * 2003-06-20 2008-10-08 Isis Pharmaceuticals Inc OLIGOMER COMPOUNDS FOR USE IN THE MODULATION OF GENES
EP1644475A4 (en) * 2003-06-20 2009-06-03 Isis Pharmaceuticals Inc DOUBLE-STRAND COMPOSITIONS WITH A 3'-ENDO-MODIFIED STRING FOR USE IN GENE MODULATION
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20070123480A1 (en) * 2003-09-11 2007-05-31 Replicor Inc. Oligonucleotides targeting prion diseases
EP1677822B1 (en) 2003-09-18 2014-04-23 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
EA009670B1 (ru) 2003-09-18 2008-02-28 Ай Эс Ай Эс ФАРМАСЬЮТИКАЛЗ, ИНК. МОДУЛИРОВАНИЕ ЭКСПРЕССИИ eIF4E
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
DK2161283T3 (da) 2003-11-17 2014-09-01 Genentech Inc SAMMENSÆTNINGER OMFATTENDE ANTISTOFFER MOD CD79b, SOM ER KONJUGERET TIL ET VÆKSTINHIBERENDE MIDDEL ELLER ET CYTOTOKSISK MIDDEL, OG FREMGANGSMÅDER TIL BEHANDLING AF TUMOR AF HÆMATOPOIETISK OPRINDELSE
JP2007520222A (ja) 2004-01-20 2007-07-26 アイシス ファーマシューティカルズ インコーポレイテッド グルココルチコイドレセプター発現の調節
US7468431B2 (en) 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US8778900B2 (en) * 2004-01-22 2014-07-15 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP1 expression
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
US8790919B2 (en) 2004-03-15 2014-07-29 Isis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of RNA by RNase H
US20050244869A1 (en) * 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
EP1737878A2 (en) 2004-04-05 2007-01-03 Alnylam Pharmaceuticals Inc. Process and reagents for oligonucleotide synthesis and purification
US20050260755A1 (en) * 2004-04-06 2005-11-24 Isis Pharmaceuticals, Inc. Sequential delivery of oligomeric compounds
US7674778B2 (en) 2004-04-30 2010-03-09 Alnylam Pharmaceuticals Oligonucleotides comprising a conjugate group linked through a C5-modified pyrimidine
JP2008500064A (ja) 2004-05-21 2008-01-10 ザ ユーエービー リサーチ ファウンデーション 可変性リンパ球受容体、その関連ポリペプチドおよび核酸、ならびにその使用
US8394947B2 (en) * 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
EP1766052A4 (en) * 2004-06-03 2009-12-16 Isis Pharmaceuticals Inc OLIGOMERIC CHIMERIC COMPOSITIONS WITH BRECH
US7427675B2 (en) 2004-08-23 2008-09-23 Isis Pharmaceuticals, Inc. Compounds and methods for the characterization of oligonucleotides
US7884086B2 (en) * 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
US7759479B1 (en) * 2004-09-13 2010-07-20 Isis Pharmaceuticals, Inc. Compositions and their uses directed to Gemin Genes
WO2006032144A1 (en) 2004-09-23 2006-03-30 Arc Pharmaceuticals, Inc. Pharmaceutical compositions and methods relating to inhibiting fibrous adhesions or inflammatory disease using fucans from various echinoderm sources
AU2005327506B2 (en) 2004-10-20 2010-07-08 Antisense Therapeutics Ltd Antisense modulation of integrin alpha4 expression
WO2006063031A2 (en) * 2004-12-06 2006-06-15 Haplomics Allelic variants of human factor viii
WO2006091905A1 (en) * 2005-02-25 2006-08-31 Gilead Sciences, Inc. Bicyclo (3.1.0) hexane derivatives as antiviral compounds
EP1869076A2 (en) 2005-03-10 2007-12-26 Genentech, Inc. Methods and compositions for modulating vascular integrity
US7476733B2 (en) * 2005-03-25 2009-01-13 The United States Of America As Represented By The Department Of Health And Human Services Development of a real-time PCR assay for detection of pneumococcal DNA and diagnosis of pneumococccal disease
US8309303B2 (en) 2005-04-01 2012-11-13 Qiagen Gmbh Reverse transcription and amplification of RNA with simultaneous degradation of DNA
JP5329949B2 (ja) 2005-05-31 2013-10-30 エコーレ ポリテクニーク フェデラーレ デ ローザンヌ 遺伝子に基づいた薬物の細胞質送達のためのトリブロックコポリマー
EP1904111A4 (en) 2005-06-03 2009-08-19 Univ Johns Hopkins COMPOSITIONS AND METHODS FOR REDUCING MICRORNA EXPRESSION FOR THE TREATMENT OF NEOPLASIA
WO2006138145A1 (en) 2005-06-14 2006-12-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
EP1929012B1 (en) 2005-08-11 2010-10-06 Synthetic Genomics, Inc. Method for in vitro recombination
EP1915461B1 (en) 2005-08-17 2018-08-01 Dx4U GmbH Composition and method for determination of ck19 expression
EP1931780B1 (en) 2005-08-29 2016-01-06 Regulus Therapeutics Inc. Antisense compounds having enhanced anti-microrna activity
CA2620856C (en) 2005-08-29 2017-11-28 Isis Pharmaceuticals, Inc. Methods for use in modulating mir-122a
EP1762627A1 (de) 2005-09-09 2007-03-14 Qiagen GmbH Verfahren zur Aktivierung einer Nukleinsäure für eine Polymerase-Reaktion
EP2189522A1 (en) 2005-10-14 2010-05-26 MUSC Foundation For Research Development Targeting PAX2 for the induction of DEFB1-mediated tumor immunity and cancer therapy
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
EP1941059A4 (en) * 2005-10-28 2010-11-03 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING THE EXPRESSION OF THE HUNTINGTIN GENE
WO2007056326A2 (en) * 2005-11-04 2007-05-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of nav1.8 gene
US20100069461A1 (en) 2005-11-09 2010-03-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor v leiden mutant gene
JP2009516710A (ja) 2005-11-21 2009-04-23 アイシス ファーマシューティカルズ, インコーポレーテッド eIF4E−BP2の発現のモジュレート
US8313901B2 (en) * 2005-12-21 2012-11-20 Yale University Methods and compositions related to the modulation of riboswitches
CN101437933B (zh) 2005-12-28 2013-11-06 斯克里普斯研究所 作为药物靶标的天然反义和非编码的rna转录物
US20070299027A1 (en) 2006-01-26 2007-12-27 Gene Hung Compositions and their uses directed to huntingtin
CN102908630B (zh) * 2006-01-27 2014-11-19 Isis制药公司 6-修饰的双环核酸类似物
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
WO2007090073A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
CA2873833A1 (en) 2006-03-31 2007-10-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 gene
BRPI0711182A2 (pt) * 2006-05-03 2011-08-23 Baltic Technology Dev agentes anti-sentido combinando base fortemente ligada-oligonucleotìdeo modificado e nuclease artificial
ES2386578T3 (es) * 2006-05-05 2012-08-23 Isis Pharmaceuticals, Inc. Compuestos y procedimientos para modular la expresión de PCSK9
DE102006020885A1 (de) * 2006-05-05 2007-11-08 Qiagen Gmbh Einführung von Sequenzelementen in Nukleinsäuren
EP2505649A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of GCGR
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
US7666854B2 (en) * 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
JP2009537153A (ja) 2006-05-19 2009-10-29 アルニラム ファーマシューティカルズ, インコーポレイテッド AhaのRNAi調節およびその治療上の使用
US7888498B2 (en) 2006-05-22 2011-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of IKK-B gene
EP2023938A4 (en) * 2006-05-23 2010-11-10 Isis Pharmaceuticals Inc MODULATION OF CHREBP EXPRESSION
WO2008011473A2 (en) 2006-07-19 2008-01-24 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hbxip
WO2008017081A1 (en) * 2006-08-04 2008-02-07 Isis Pharmaceuticals, Inc. Compositions and methods for the modulation of jnk proteins
WO2008033866A2 (en) * 2006-09-11 2008-03-20 Yale University Methods and compositions for the use of lysine riboswitches
US7947487B2 (en) 2006-10-05 2011-05-24 Massachusetts Institute Of Technology Multifunctional encoded particles for high-throughput analysis
DK2410054T4 (da) 2006-10-18 2020-02-10 Ionis Pharmaceuticals Inc Antisenseforbindelser
US8999317B2 (en) 2006-11-01 2015-04-07 University Of Rochester Methods and compositions related to the structure and function of APOBEC3G
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
US8084437B2 (en) * 2006-11-27 2011-12-27 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
CN101600451A (zh) * 2006-12-11 2009-12-09 犹他大学研究基金会 用于治疗病理性血管生成和血管通透性的组合物和方法
EP2097448A4 (en) 2006-12-22 2010-07-21 Univ Utah Res Found PROCEDURE FOR THE EVIDENCE OF EYE DISEASES AND SUFFERINGS AND THEIR TREATMENT
EP2121987B1 (en) 2007-02-09 2012-06-13 Northwestern University Particles for detecting intracellular targets
MX2009009127A (es) * 2007-02-27 2009-10-19 Univ Northwestern Unión de moléculas a nanopartículas.
EP2471925A1 (en) 2007-03-22 2012-07-04 Yale University Methods and compositions related to riboswitches that control alternative splicing
WO2008121604A2 (en) 2007-03-29 2008-10-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from the ebola
AU2008251381B2 (en) 2007-05-11 2014-10-30 The Johns Hopkins University Biomarkers for melanoma
EP2426218A1 (en) 2007-05-29 2012-03-07 Yale University Riboswitches and methods and compositions for use of and with riboswitches
KR20100017893A (ko) * 2007-05-29 2010-02-16 예일 유니버시티 택일적 스플라이싱 및 rna 프로세싱을 조절하는 리보스위치와 관련된 방법 및 조성물
DK2160464T3 (da) 2007-05-30 2014-08-04 Univ Northwestern Nukleinsyrefunktionaliserede nanopartikler til terapeutiske anvendelser
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US7807372B2 (en) * 2007-06-04 2010-10-05 Northwestern University Screening sequence selectivity of oligonucleotide-binding molecules using nanoparticle based colorimetric assay
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
ATE538127T1 (de) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
AU2008286771B2 (en) 2007-08-15 2013-08-15 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
WO2009032702A2 (en) 2007-08-28 2009-03-12 Uab Research Foundation Synthetic apolipoprotein e mimicking polypeptides and methods of use
US9422363B2 (en) 2007-08-28 2016-08-23 Uab Research Foundation Synthetic apolipoprotein E mimicking polypeptides and methods of use
US8445217B2 (en) 2007-09-20 2013-05-21 Vanderbilt University Free solution measurement of molecular interactions by backscattering interferometry
US7951785B2 (en) 2007-09-21 2011-05-31 California Institute Of Technology NFIA in glial fate determination, glioma therapy and astrocytoma treatment
CA2704560A1 (en) * 2007-11-05 2009-05-14 Baltic Technology Development, Ltd. Use of oligonucleotides with modified bases in hybridization of nucleic acids
WO2009067243A2 (en) 2007-11-20 2009-05-28 Isis Pharmaceuticals Inc. Modulation of cd40 expression
EP2848688A1 (en) 2007-12-10 2015-03-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor VII gene
EP3100718B1 (en) 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids
EP2265627A2 (en) * 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
AU2009213147A1 (en) 2008-02-11 2009-08-20 Rxi Pharmaceuticals Corp. Modified RNAi polynucleotides and uses thereof
CN102014932B (zh) 2008-03-05 2015-11-25 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的组合物和方法
US8426378B2 (en) 2008-03-21 2013-04-23 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucelosides and methods for their use
US8846639B2 (en) * 2008-04-04 2014-09-30 Isis Pharmaceutical, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
DK2285819T3 (da) * 2008-04-04 2013-12-02 Isis Pharmaceuticals Inc Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider
ES2686708T3 (es) 2008-04-18 2018-10-19 Baxter International Inc. Composición basada en microesferas para prevenir y/o revertir la diabetes autoinmune de nueva aparición
US20090274696A1 (en) * 2008-04-29 2009-11-05 Wyeth Methods for treating inflammation
US8222221B2 (en) 2008-06-04 2012-07-17 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small RNA targeting of gene promoters
JP5438922B2 (ja) * 2008-06-25 2014-03-12 日東電工株式会社 核酸の製造方法
BRPI0916440A2 (pt) * 2008-07-15 2018-06-05 Hoffmann La Roche composições e métodos para inibir a expressão dos genes receptores tgf-beta
US8815818B2 (en) 2008-07-18 2014-08-26 Rxi Pharmaceuticals Corporation Phagocytic cell delivery of RNAI
JP2011529686A (ja) 2008-07-29 2011-12-15 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム ポリグルタミンタンパク質発現の選択的阻害
US20110237646A1 (en) * 2008-08-07 2011-09-29 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
WO2010027832A1 (en) 2008-08-25 2010-03-11 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to ctgf
US8252762B2 (en) 2008-08-25 2012-08-28 Excaliard Pharmaceuticals, Inc. Antisense oligonucleotides directed against connective tissue growth factor and uses thereof
EP3208337A1 (en) 2008-09-02 2017-08-23 Alnylam Pharmaceuticals, Inc. Compositions for combined inhibition of mutant egfr and il-6 expression
CN102405286A (zh) 2008-09-22 2012-04-04 阿克赛医药公司 减小大小的自递送RNAi化合物
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
WO2010036696A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
EP3584320A1 (en) 2008-09-25 2019-12-25 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
EP2350043B9 (en) 2008-10-09 2014-08-20 TEKMIRA Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
EP2379084B1 (en) 2008-10-15 2017-11-22 Ionis Pharmaceuticals, Inc. Modulation of factor 11 expression
CA3222620A1 (en) 2008-10-20 2010-04-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
EP2447274B1 (en) 2008-10-24 2017-10-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds and methods
US8883752B2 (en) 2008-10-24 2014-11-11 Isis Pharmaceuticals, Inc. 5′ and 2′ BIS-substituted nucleosides and oligomeric compounds prepared therefrom
CN102216457A (zh) * 2008-11-17 2011-10-12 霍夫曼-拉罗奇有限公司 抑制凝血因子vii基因的表达的组合物和方法
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
CA2744207C (en) 2008-11-24 2019-05-28 Northwestern University Polyvalent rna-nanoparticle compositions
KR101829469B1 (ko) 2008-12-04 2018-03-30 큐알엔에이, 인크. Epo에 대한 천연 안티센스 전사체의 억제에 의해 에리트로포에틴(epo) 관련된 질환의 치료
CA2746001C (en) 2008-12-04 2020-03-31 Joseph Collard Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirtuin 1
RU2746478C2 (ru) 2008-12-04 2021-04-14 КьюРНА, Инк. Лечение связанных с геном-супрессором опухолей заболеваний посредством ингибирования природного транскрипта в антисмысловой ориентации относительно этого гена
AU2009324534B2 (en) 2008-12-10 2015-07-30 Alnylam Pharmaceuticals, Inc. GNAQ targeted dsRNA compositions and methods for inhibiting expression
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
EP2385760A4 (en) * 2009-01-08 2015-09-30 Univ Northwestern INHIBITION OF BACTERIAL PROTEIN PRODUCTION WITH POLYVALENT OLIGONUCLEOTIDE-MODIFIED NANOPARTICLE CONJUGATES
KR101546673B1 (ko) * 2009-01-15 2015-08-25 삼성전자주식회사 전자 사진용 토너 및 그의 제조방법
US20120101148A1 (en) 2009-01-29 2012-04-26 Alnylam Pharmaceuticals, Inc. lipid formulation
CN102307996A (zh) * 2009-02-03 2012-01-04 弗·哈夫曼-拉罗切有限公司 用于抑制ptp1b基因表达的组合物和方法
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US8536320B2 (en) 2009-02-06 2013-09-17 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
US20110319476A1 (en) 2009-02-12 2011-12-29 Opko Curna, Llc Treatment of glial cell derived neurotrophic factor (gdnf) related diseases by inhibition of natural antisense transcript to gdnf
EP2396038B1 (en) 2009-02-12 2015-10-21 CuRNA, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
JP6250263B2 (ja) 2009-03-04 2017-12-20 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sirt1関連疾患の治療
CA2754043A1 (en) 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
EP2408919B1 (en) 2009-03-16 2017-10-18 CuRNA, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
ES2627763T3 (es) 2009-03-17 2017-07-31 Curna, Inc. Tratamiento de enfermedades relacionadas con el homólogo tipo delta 1 (dlk1) por inhibición de transcrito antisentido natural a dlk1
WO2010120420A1 (en) 2009-04-15 2010-10-21 Northwestern University Delivery of oligonucleotide-functionalized nanoparticles
EP3248618A1 (en) 2009-04-22 2017-11-29 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
EP2421972A2 (en) 2009-04-24 2012-02-29 The Board of Regents of The University of Texas System Modulation of gene expression using oligomers that target gene regions downstream of 3' untranslated regions
WO2010127195A2 (en) 2009-05-01 2010-11-04 Curna, Inc. Antisense oligonucleotides of hemoglobins
WO2010129687A1 (en) 2009-05-05 2010-11-11 Alnylam Pharmaceuticals, Inc Methods of delivering oligonucleotides to immune cells
KR20240015723A (ko) 2009-05-05 2024-02-05 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
WO2010129746A2 (en) 2009-05-06 2010-11-11 Curna, Inc. Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
CN102459596B (zh) 2009-05-06 2016-09-07 库尔纳公司 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病
AU2010247389A1 (en) * 2009-05-15 2011-10-27 F. Hoffmann-La Roche Ag Compositions and methods for inhibiting expression of glucocorticoid receptor (GCR) genes
WO2010132665A1 (en) 2009-05-15 2010-11-18 Yale University Gemm riboswitches, structure-based compound design with gemm riboswitches, and methods and compositions for use of and with gemm riboswitches
DK2432881T3 (en) 2009-05-18 2018-02-26 Curna Inc TREATMENT OF REPROGRAMMING FACTOR-RELATED DISEASES BY INHIBITING NATURAL ANTISENSE TRANSCRIPTS TO A REPROGRAMMING FACTOR
KR101703695B1 (ko) 2009-05-22 2017-02-08 큐알엔에이, 인크. 전사 인자 e3(tfe3)에 대한 천연 안티센스 전사체의 억제에 의해 tfe3 및 인슐린 수용체 기질 2(irs2)의 치료
ES2618576T3 (es) 2009-05-28 2017-06-21 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen antivírico mediante la inhibición de una transcripción antisentido natural a un gen antivírico
CA2764609C (en) 2009-06-10 2018-10-02 Alnylam Pharmaceuticals, Inc. Improved cationic lipid of formula i
CA2765509C (en) 2009-06-16 2021-08-17 Joseph Collard Treatment of paraoxonase 1 (pon1) related diseases by inhibition of natural antisense transcript to pon1
JP5944311B2 (ja) 2009-06-16 2016-07-05 クルナ・インコーポレーテッド コラーゲン遺伝子に対する天然アンチセンス転写物の抑制によるコラーゲン遺伝子関連疾患の治療
CN115227710A (zh) 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
KR101807323B1 (ko) 2009-06-24 2017-12-08 큐알엔에이, 인크. Tnfr2에 대한 천연 안티센스 전사체의 억제에 의한 종양 괴사 인자 수용체 2(tnfr2) 관련된 질환의 치료
US8921330B2 (en) 2009-06-26 2014-12-30 Curna, Inc. Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
WO2011017516A2 (en) 2009-08-05 2011-02-10 Curna, Inc. Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011020023A2 (en) 2009-08-14 2011-02-17 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
US20120157324A1 (en) 2009-08-17 2012-06-21 Yale University Methylation biomarkers and methods of use
WO2011031482A2 (en) 2009-08-25 2011-03-17 Curna, Inc. Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
ES2599076T3 (es) 2009-09-02 2017-01-31 Genentech, Inc. Smoothened mutante y métodos de utilización del mismo
WO2011050194A1 (en) 2009-10-22 2011-04-28 Genentech, Inc. Methods and compositions for modulating hepsin activation of macrophage-stimulating protein
US20110129832A1 (en) * 2009-10-27 2011-06-02 Swift Biosciences, Inc. Polynucleotide Primers and Probes
KR20120136345A (ko) 2009-10-30 2012-12-18 노오쓰웨스턴 유니버시티 주형화된 나노컨쥬게이트
US20110110860A1 (en) 2009-11-02 2011-05-12 The Board Of Regents Of The University Of Texas System Modulation of ldl receptor gene expression with double-stranded rnas targeting the ldl receptor gene promoter
WO2011056215A1 (en) 2009-11-03 2011-05-12 Landers James P Versatile, visible method for detecting polymeric analytes
US20110112176A1 (en) 2009-11-09 2011-05-12 John Frederick Boylan Compositions and methods for inhibiting expression of kif10 genes
JP2013511285A (ja) 2009-11-23 2013-04-04 スイフト・バイオサイエンシズ・インコーポレイテツド 一本鎖標的分子を伸長させるデバイス
CN102741294A (zh) 2009-11-30 2012-10-17 霍夫曼-拉罗奇有限公司 治疗和诊断表达slc34a2(tat211=seqid2)的肿瘤的抗体
WO2011068978A1 (en) 2009-12-02 2011-06-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Methanocarba adenosine derivatives and dendrimer conjugates thereof
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
US20110152349A1 (en) 2009-12-18 2011-06-23 Anke Geick Compositions and methods for inhibiting expression of il-18 genes
US9068183B2 (en) 2009-12-23 2015-06-30 Curna, Inc. Treatment of uncoupling protein 2 (UCP2) related diseases by inhibition of natural antisense transcript to UCP2
CN102869776B (zh) 2009-12-23 2017-06-23 库尔纳公司 通过抑制肝细胞生长因子(hgf)的天然反义转录物而治疗hgf相关疾病
EP2519633B1 (en) 2009-12-29 2017-10-25 CuRNA, Inc. Treatment of nuclear respiratory factor 1 (nrf1) related diseases by inhibition of natural antisense transcript to nrf1
JP5982288B2 (ja) 2009-12-29 2016-08-31 カッパーアールエヌエー,インコーポレイテッド 腫瘍タンパク質63(p63)に対する天然アンチセンス転写物の阻害による腫瘍タンパク質63関連疾患の治療
KR101878501B1 (ko) 2010-01-04 2018-08-07 큐알엔에이, 인크. 인터페론 조절 인자 8 (irf8)에 대한 자연 안티센스 전사체의 저해에 의한 인터페론 조절 인자 8 (irf8) 관련된 질환의 치료
WO2011085066A2 (en) 2010-01-06 2011-07-14 Curna, Inc. Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
WO2011085102A1 (en) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
CN102803493B (zh) 2010-01-11 2018-07-31 库尔纳公司 通过抑制性激素结合球蛋白(shbg)的天然反义转录物而治疗shbg相关疾病
EP2524042A2 (en) 2010-01-12 2012-11-21 Yale University Structured rna motifs and compounds and methods for their use
BR112012017483A2 (pt) * 2010-01-14 2019-09-24 Haplomics Inc previsão e redução de aloimunogenicidade de terapêuticos de proteína
KR101853510B1 (ko) 2010-01-25 2018-06-20 큐알엔에이, 인크. Rnase h1에 대한 천연 안티센스 전사체의 억제에 의한 rnase h1과 관련된 질환의 치료
US9574191B2 (en) 2010-02-03 2017-02-21 The Board Of Regents Of The University Of Texas System Selective inhibition of polyglutamine protein expression
CA2824843A1 (en) 2010-02-04 2011-08-11 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
TW201129365A (en) * 2010-02-05 2011-09-01 Hoffmann La Roche Compositions and methods for inhibiting expression of IKK2 genes
CA2790506A1 (en) 2010-02-22 2011-08-25 Curna, Inc. Treatment of pyrroline-5-carboxylate reductase 1 (pycr1) related diseases by inhibition of natural antisense transcript to pycr1
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
WO2011106297A2 (en) 2010-02-23 2011-09-01 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011105902A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-beta (c8-beta) and uses thereof
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
US20130101512A1 (en) 2010-03-12 2013-04-25 Chad A. Mirkin Crosslinked polynucleotide structure
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
KR20180044433A (ko) 2010-03-24 2018-05-02 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 rna 간섭
CN103200945B (zh) 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
ES2893199T3 (es) 2010-03-29 2022-02-08 Alnylam Pharmaceuticals Inc Terapia de ARNbc para amiloidosis ocular relacionada con transtiretina (TTR)
US8507663B2 (en) 2010-04-06 2013-08-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of CD274/PD-L1 gene
EP2556160A4 (en) 2010-04-09 2013-08-21 Curna Inc TREATMENT OF ILLNESSES ASSOCIATED WITH FIBROBLASTIC GROWTH FACTOR 21 (FGF21) BY INHIBITION OF THE NATURAL ANTISENSE TRANSCRIPT AGAINST FGF21
WO2011133695A2 (en) 2010-04-20 2011-10-27 Swift Biosciences, Inc. Materials and methods for nucleic acid fractionation by solid phase entrapment and enzyme-mediated detachment
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
US9127033B2 (en) 2010-04-28 2015-09-08 Isis Pharmaceuticals, Inc. 5′ modified nucleosides and oligomeric compounds prepared therefrom
KR20180026798A (ko) 2010-04-29 2018-03-13 아이오니스 파마수티컬즈, 인코포레이티드 트랜스티레틴 발현의 조절
WO2011139387A1 (en) 2010-05-03 2011-11-10 Opko Curna, Llc Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt)
AU2011248354A1 (en) 2010-05-03 2012-11-08 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
US20130203045A1 (en) 2010-05-26 2013-08-08 University Of Virginia Patent Foundation Method for detecting nucleic acids based on aggregate formation
KR101857090B1 (ko) 2010-05-26 2018-06-26 큐알엔에이, 인크. 무조(無調)의 동소체 1 (atoh1)에 대한 자연 안티센스 전사체의 저해에 의한 atoh1 관련된 질환의 치료
WO2011153323A2 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
WO2011156432A2 (en) 2010-06-07 2011-12-15 Firefly Bioworks, Inc. Scanning multifunctional particles
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156202A1 (en) 2010-06-08 2011-12-15 Isis Pharmaceuticals, Inc. Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156713A1 (en) 2010-06-11 2011-12-15 Vanderbilt University Multiplexed interferometric detection system and method
US9168297B2 (en) 2010-06-23 2015-10-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Regulation of skin pigmentation by neuregulin-1 (NRG-1)
RU2611190C2 (ru) 2010-07-14 2017-02-21 Курна, Инк. Лечение заболеваний, связанных с геном dlg, путем ингибирования природного антисмыслового транскрипта гена dlg
EP3031920B1 (en) 2010-07-19 2019-08-21 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (dmpk) expression
US20130143955A1 (en) 2010-08-09 2013-06-06 Yale University Cyclic di-GMP-II Riboswitches, Motifs, and Compounds, and Methods for Their Use
DK2625197T3 (en) 2010-10-05 2016-10-03 Genentech Inc Smoothened MUTANT 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
WO2012052258A1 (en) 2010-10-18 2012-04-26 Arrowhead Research Corporation Compositions and methods for inhibiting expression of rrm2 genes
EP2630241B1 (en) 2010-10-22 2018-10-17 CuRNA, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
CN103201387B (zh) 2010-10-27 2018-02-02 库尔纳公司 通过抑制干扰素相关发育调节因子1(ifrd1)的天然反义转录物而治疗ifrd1相关疾病
US9339513B2 (en) 2010-11-09 2016-05-17 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
CA2817960C (en) 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
RU2608493C2 (ru) 2010-11-23 2017-01-18 Курна, Инк. Лечение заболеваний, связанных с nanog, путем ингибирования природного антисмыслового транскрипта nanog
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
EP2670404B1 (en) 2011-02-02 2018-08-29 The Trustees of Princeton University Sirtuin modulators as virus production modulators
PT2670411T (pt) 2011-02-02 2019-06-18 Excaliard Pharmaceuticals Inc Compostos anti sentido visando um fator de crescimento do tecido conetivo (ctfg) para utilização num método de tratamento de queloides ou cicatrizes hipertróficas
WO2012106591A1 (en) 2011-02-03 2012-08-09 Mirna Therapeutics, Inc. Synthetic mimics of mir-34
JP2014506789A (ja) 2011-02-03 2014-03-20 マーナ セラピューティクス インコーポレイテッド miR−124の合成模倣体
WO2012109395A1 (en) 2011-02-08 2012-08-16 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US9562853B2 (en) 2011-02-22 2017-02-07 Vanderbilt University Nonaqueous backscattering interferometric methods
CN103813810B (zh) 2011-03-29 2021-08-03 阿尔尼拉姆医药品有限公司 用于抑制tmprss6基因表达的组合物和方法
EP3460064B8 (en) 2011-04-03 2024-03-20 The General Hospital Corporation d/b/a Massachusetts General Hospital Efficient protein expression in vivo using modified rna (mod-rna)
US20140186844A1 (en) 2011-04-26 2014-07-03 Swift Biosciences, Inc. Polynucleotide primers and probes
WO2012151268A1 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system for high throughput optical and label free detection of analytes
WO2012151289A2 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system to detect aggregate formation on a substrate
WO2012170347A1 (en) 2011-06-09 2012-12-13 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
US9593330B2 (en) 2011-06-09 2017-03-14 Curna, Inc. Treatment of frataxin (FXN) related diseases by inhibition of natural antisense transcript to FXN
CA2838984A1 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
CN120555426A (zh) 2011-06-21 2025-08-29 阿尔尼拉姆医药品有限公司 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法
KR102540778B1 (ko) 2011-06-21 2023-06-07 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
US9068184B2 (en) 2011-06-21 2015-06-30 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein C (PROC) genes
WO2012178033A2 (en) 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
WO2013003808A1 (en) 2011-06-29 2013-01-03 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
KR102167524B1 (ko) 2011-06-30 2020-10-20 애로우헤드 파마슈티컬스 인코포레이티드 B형 간염 바이러스의 유전자 발현 저해용 조성물 및 방법
JP2014526887A (ja) 2011-08-01 2014-10-09 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 造血幹細胞移植の成功率を改善する方法
EP3556859B1 (en) 2011-08-11 2021-04-07 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
CA2847698C (en) 2011-09-14 2020-09-01 Northwestern University Nanoconjugates able to cross the blood-brain barrier
US9580713B2 (en) 2011-09-17 2017-02-28 Yale University Fluoride-responsive riboswitches, fluoride transporters, and methods of use
HUE039133T2 (hu) 2011-10-14 2018-12-28 Hoffmann La Roche Anti-HtrA1 antitestek és felhasználási módszerek
WO2013059740A1 (en) 2011-10-21 2013-04-25 Foundation Medicine, Inc. Novel alk and ntrk1 fusion molecules and uses thereof
PL2791160T3 (pl) 2011-12-16 2022-06-20 Modernatx, Inc. Kompozycje zmodyfikowanego mrna
DK2794880T3 (en) 2011-12-22 2018-07-23 Ionis Pharmaceuticals Inc METHOD OF MODULATING METASTASE-ASSOCIATED-IN-LONG-ADENOCARCINOM TRANSCRIPT-1- (MALATE-1) EXPRESSION
CA2860731C (en) 2012-01-10 2023-02-28 M. Mahmood Hussain Method of treating hyperlipidemia and atherosclerosis with mir-30c
WO2013120003A1 (en) 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
EP2825672B1 (en) 2012-03-13 2019-02-13 Swift Biosciences, Inc. Methods and compositions for size-controlled homopolymer tailing of substrate polynucleotides by a nucleic acid polymerase
ES2694592T3 (es) 2012-03-15 2018-12-21 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado del cerebro (BDNF) por inhibición del transcrito antisentido natural de BDNF
CA2907072A1 (en) 2012-03-16 2013-09-19 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of dmpk
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
HK1206779A1 (en) 2012-04-02 2016-01-15 Modernatx, Inc. Modified polynucleotides for the production of nuclear proteins
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
WO2013159108A2 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US9273949B2 (en) 2012-05-11 2016-03-01 Vanderbilt University Backscattering interferometric methods
US9518261B2 (en) 2012-05-22 2016-12-13 Ionis Pharmaceuticals, Inc. Modulation of enhancer RNA mediated gene expression
PL3461895T3 (pl) 2012-06-25 2021-01-11 Ionis Pharmaceuticals, Inc. Modulacja ekspresji ube3a-ats
US20140038182A1 (en) 2012-07-17 2014-02-06 Dna Logix, Inc. Cooperative primers, probes, and applications thereof
EP2877579B1 (en) 2012-07-27 2019-12-18 Ionis Pharmaceuticals, Inc. Modulation of renin-angiotensin system (ras) related diseases by angiotensinogen
EP2885312B1 (en) 2012-08-15 2025-09-03 Ionis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified capping protocols
US9993522B2 (en) 2012-09-18 2018-06-12 Uti Limited Partnership Treatment of pain by inhibition of USP5 de-ubiquitinase
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
US9695418B2 (en) 2012-10-11 2017-07-04 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and uses thereof
WO2014059364A1 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Methods of treating kennedy's disease
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
CA2887702C (en) 2012-10-26 2023-08-01 Geron Corporation C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders
AU2013337277B2 (en) 2012-11-05 2018-03-08 Foundation Medicine, Inc. Novel NTRK1 fusion molecules and uses thereof
ES2949394T3 (es) 2012-11-05 2023-09-28 Found Medicine Inc Moléculas de fusión novedosas y usos de las mismas
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
AU2013374345A1 (en) 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
CA3150658A1 (en) 2013-01-18 2014-07-24 Foundation Medicine, Inc. Methods of treating cholangiocarcinoma
DK2951191T3 (en) 2013-01-31 2019-01-14 Ionis Pharmaceuticals Inc PROCEDURE FOR MANUFACTURING OLIGOMERIC COMPOUNDS USING MODIFIED CLUTCH PROTOCOLS
KR102169899B1 (ko) 2013-02-14 2020-10-26 아이오니스 파마수티컬즈, 인코포레이티드 지질단백질 리파제 결핍 (lpld) 모집단에서 아포지질단백질 c-iii (apociii) 발현의 조절
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
US10398661B2 (en) 2013-02-28 2019-09-03 The Board Of Regents Of The University Of Texas System Methods for classifying a cancer as susceptible to TMEPAI-directed therapies and treating such cancers
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
CN120574828A (zh) 2013-03-14 2025-09-02 阿尔尼拉姆医药品有限公司 补体组分C5 iRNA组合物及其使用方法
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
CN115261411A (zh) 2013-04-04 2022-11-01 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
CN107573333B (zh) * 2013-04-10 2019-10-18 江苏恒瑞医药股份有限公司 替格瑞洛的中间体及其制备方法和替格瑞洛的制备方法
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
PL2999785T3 (pl) 2013-05-22 2018-09-28 Alnylam Pharmaceuticals, Inc. Kompozycje iRNA Serpina1 i sposoby ich zastosowania
SG10202103166XA (en) 2013-05-22 2021-04-29 Alnylam Pharmaceuticals Inc Tmprss6 irna compositions and methods of use thereof
EP3004396B1 (en) 2013-06-06 2019-10-16 The General Hospital Corporation Compositions for the treatment of cancer
DK3007704T3 (da) 2013-06-13 2021-03-29 Antisense Therapeutics Ltd Kombinationsterapi til akromegali
US9909124B2 (en) 2013-06-21 2018-03-06 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein C-III expression for improving a diabetic profile
RU2700244C2 (ru) 2013-07-02 2019-09-13 Ионис Фармасьютикалз, Инк. Модуляторы рецептора гормона роста
PT3019619T (pt) 2013-07-11 2021-11-11 Modernatx Inc Composições que compreendem polinucleótidos sintéticos que codificam proteínas relacionadas com crispr e sgarn sintéticos e métodos de utilização
HRP20200250T1 (hr) 2013-08-08 2020-05-29 The Scripps Research Institute Metoda site-specifično enzimsko obilježavanje nukleinskih kiselina in vitro ugradnjom neprirodnih nukleotida
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
JP6652922B2 (ja) 2013-08-28 2020-02-26 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. プレカリクレイン(pkk)発現の調節
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160222389A1 (en) 2013-09-13 2016-08-04 Ionis Pharmaceuticals, Inc. Modulators of complement factor b
CA2925107A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
WO2015051214A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
CN105683163B (zh) 2013-10-04 2018-11-09 诺华股份有限公司 RNA干扰中使用的RNAi剂的3’端帽
EP3052628B1 (en) 2013-10-04 2020-02-26 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
WO2015050871A2 (en) 2013-10-04 2015-04-09 Novartis Ag Organic compounds to treat hepatitis b virus
WO2015051366A2 (en) 2013-10-04 2015-04-09 Novartis Ag Novel formats for organic compounds for use in rna interference
US10584387B2 (en) 2013-10-09 2020-03-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Detection of hepatitis delta virus (HDV) for the diagnosis and treatment of Sjögren's syndrome and lymphoma
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
WO2015061246A1 (en) 2013-10-21 2015-04-30 Isis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
CA2928779A1 (en) 2013-10-21 2015-04-30 The General Hospital Corporation Methods relating to circulating tumor cell clusters and the treatment of cancer
WO2015066708A1 (en) 2013-11-04 2015-05-07 Northwestern University Quantification and spatio-temporal tracking of a target using a spherical nucleic acid (sna)
CN113151180A (zh) 2013-12-02 2021-07-23 菲奥医药公司 癌症的免疫治疗
CN112107693B (zh) 2013-12-03 2023-05-26 西北大学 脂质体颗粒、制备所述脂质体颗粒的方法以及其用途
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
US10385388B2 (en) 2013-12-06 2019-08-20 Swift Biosciences, Inc. Cleavable competitor polynucleotides
SG11201604692UA (en) 2013-12-12 2016-07-28 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
JP6599334B2 (ja) 2013-12-20 2019-10-30 ザ ジェネラル ホスピタル コーポレイション 血中循環腫瘍細胞に関する方法およびアッセイ
US20170037409A1 (en) 2013-12-24 2017-02-09 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
CA2937539A1 (en) 2014-02-04 2015-08-13 Genentech, Inc. Mutant smoothened and methods of using the same
CN106103718B (zh) 2014-02-11 2021-04-02 阿尔尼拉姆医药品有限公司 己酮糖激酶(KHK)iRNA组合物及其使用方法
WO2015142910A1 (en) * 2014-03-17 2015-09-24 Isis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
RU2019130898A (ru) 2014-03-19 2019-11-11 Ионис Фармасьютикалз, Инк. Композиции для модуляции экспрессии атаксина 2
WO2015143245A1 (en) 2014-03-19 2015-09-24 Isis Pharmaceuticals, Inc. Methods for modulating ataxin 2 expression
SG10201910844SA (en) 2014-04-01 2020-01-30 Biogen Ma Inc Compositions for modulating sod-1 expression
TWI638047B (zh) 2014-04-09 2018-10-11 史基普研究協會 藉由核酸三磷酸酯轉運子將非天然或經修飾的核苷三磷酸酯輸入至細胞中
MA39835A (fr) 2014-04-17 2017-02-22 Biogen Ma Inc Compositions et procédés de modulation de l'épissage du smn2 chez un patient
WO2015164693A1 (en) 2014-04-24 2015-10-29 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
WO2015168108A2 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic targeting mdm2 or mycn
KR20200102553A (ko) 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
WO2015168532A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating pkk expression
DK3137596T3 (da) 2014-05-01 2019-09-02 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af komplement faktor b-ekspression
AU2015252895B2 (en) 2014-05-01 2021-07-15 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
EP3137115B1 (en) 2014-05-01 2020-10-14 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療serpinc1相關疾患之方法和組成物
WO2015179724A1 (en) 2014-05-22 2015-11-26 Alnylam Pharmaceuticals, Inc. Angiotensinogen (agt) irna compositions and methods of use thereof
JP2017518307A (ja) 2014-06-02 2017-07-06 チルドレンズ メディカル センター コーポレーション 免疫調節のための方法および組成物
US10434064B2 (en) 2014-06-04 2019-10-08 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
JP6851201B2 (ja) 2014-06-10 2021-03-31 エラスムス ユニバーシティ メディカルセンター ロッテルダムErasmus University Medical Center Rotterdam ポンペ病の治療に有用なアンチセンスオリゴヌクレオチド
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α-1抗胰蛋白酶基因表現之組合物及方法
AU2015289583A1 (en) 2014-07-16 2017-02-02 Modernatx, Inc. Chimeric polynucleotides
US9951327B1 (en) 2014-07-17 2018-04-24 Integrated Dna Technologies, Inc. Efficient and rapid method for assembling and cloning double-stranded DNA fragments
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
USRE50320E1 (en) 2014-07-31 2025-03-04 Uab Research Foundation APOE mimetic peptides and higher potency to clear plasma cholesterol
AU2015305482B2 (en) 2014-08-19 2021-04-01 Northwestern University Protein/oligonucleotide core-shell nanoparticle therapeutics
KR20170070022A (ko) 2014-08-29 2017-06-21 칠드런'즈 메디컬 센터 코포레이션 암의 치료를 위한 방법 및 조성물
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
US10060921B2 (en) 2014-08-29 2018-08-28 Alnylam Pharmaceuticals, Inc. Methods of treating transthyretin (TTR) mediated amyloidosis
KR102689262B1 (ko) 2014-09-05 2024-07-30 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
EA201790642A1 (ru) 2014-09-18 2017-09-29 Ти Юниверсити Оф Бритиш Коламбиа Аллель-специфическая терапия для гаплотипов болезни хантингтона
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3904519A1 (en) 2014-10-30 2021-11-03 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
CR20170191A (es) 2014-11-10 2017-09-29 Glaxosmithkline Intellectual Property (No 2) Ltd Composiciones farmacéuticas de acción prolongada para la hepatitis c
CA2967184A1 (en) 2014-11-10 2016-05-19 Glaxosmithkline Intellectual Property (No.2) Limited Combination long acting compositions and methods for hepatitis c
JP2017535552A (ja) 2014-11-17 2017-11-30 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. アポリポタンパク質C3(APOC3)iRNA組成物およびその使用方法
WO2016081911A2 (en) 2014-11-21 2016-05-26 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
US10400243B2 (en) 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
AU2015364508A1 (en) 2014-12-18 2017-07-06 Alnylam Pharmaceuticals, Inc. ReversirTM compounds
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016115490A1 (en) 2015-01-16 2016-07-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
WO2016118812A1 (en) 2015-01-23 2016-07-28 Vanderbilt University A robust interferometer and methods of using same
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
WO2016137923A1 (en) 2015-02-23 2016-09-01 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
BR112017017178A2 (pt) 2015-02-26 2018-04-03 Ionis Pharmaceuticals Inc moduladores específicos de alelo de rodopsina de p23h
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
JP2018510657A (ja) 2015-03-27 2018-04-19 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 改変t細胞ならびにそれを作製および使用する方法
CN107429250B (zh) 2015-04-03 2022-03-01 Ionis制药公司 用于调节tmprss6表达的化合物和方法
WO2016164746A1 (en) 2015-04-08 2016-10-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
US10407678B2 (en) 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
PT3283080T (pt) 2015-04-16 2020-06-08 Ionis Pharmaceuticals Inc Composições para modular a expressão de c9orf72
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
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
CN108135923B (zh) 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1(sod1)的核酸分子
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
EP3324980B1 (en) 2015-07-17 2021-11-10 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
WO2017021961A1 (en) 2015-08-04 2017-02-09 Yeda Research And Development Co. Ltd. Methods of screening for riboswitches and attenuators
AU2016306275A1 (en) 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
AU2016315584B2 (en) 2015-09-02 2022-07-14 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
PT3353303T (pt) 2015-09-25 2023-10-10 Academisch Ziekenhuis Leiden Composições e métodos para modulação da expressão de ataxina 3
JP2018530325A (ja) 2015-10-08 2018-10-18 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. アンジオテンシノーゲンの発現を調節するための化合物及び方法
EP3365446A4 (en) 2015-10-19 2019-06-26 Phio Pharmaceuticals Corp. SMALL SELF-LEANING NUCLEIC ACID COMPOUNDS TURNED AGAINST LONG NON-CODING RNA
EP3368578B1 (en) 2015-10-30 2021-03-17 H. Hoffnabb-La Roche Ag Anti-htra1 antibodies and methods of use thereof
AU2016349954B2 (en) 2015-11-05 2022-08-25 Antisense Therapeutics Ltd Mobilizing leukemia cells
CA2999341A1 (en) 2015-11-06 2017-05-11 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
WO2017096395A1 (en) 2015-12-04 2017-06-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
AU2015416656B2 (en) 2015-12-07 2023-02-23 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for Pompe disease
TWI836281B (zh) 2015-12-07 2024-03-21 美商健贊公司 治療serpinc1相關疾患之方法及組成物
US11761007B2 (en) 2015-12-18 2023-09-19 The Scripps Research Institute Production of unnatural nucleotides using a CRISPR/Cas9 system
CA3006015A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
AU2017205462A1 (en) 2016-01-05 2018-06-07 Ionis Pharmaceuticals, Inc. Methods for reducing LRRK2 expression
US10627396B2 (en) 2016-01-29 2020-04-21 Vanderbilt University Free-solution response function interferometry
WO2017136558A1 (en) 2016-02-04 2017-08-10 Curis, Inc. Mutant smoothened and methods of using the same
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
AU2017234678A1 (en) 2016-03-16 2018-08-16 Ionis Pharmaceuticals, Inc. Methods of modulating KEAP1
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
WO2017214518A1 (en) 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
CA3023514A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
US20190218257A1 (en) 2016-06-24 2019-07-18 The Scripps Research Institute Novel nucleoside triphosphate transporter and uses thereof
CN109844103A (zh) 2016-07-21 2019-06-04 美克斯细胞有限公司 用于修饰基因组dna的方法和组合物
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
WO2018039629A2 (en) 2016-08-25 2018-03-01 Northwestern University Micellar spherical nucleic acids from thermoresponsive, traceless templates
WO2018055577A1 (en) 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
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
WO2018102745A1 (en) 2016-12-02 2018-06-07 Cold Spring Harbor Laboratory Modulation of lnc05 expression
KR20190098181A (ko) 2016-12-16 2019-08-21 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물을 사용하여 TTR-관련 질병을 치료하거나 예방하는 방법
KR102675026B1 (ko) 2017-01-10 2024-06-17 애로우헤드 파마슈티컬스 인코포레이티드 알파-1 항트립신 (AAT) RNAi 작용제, AAT RNAi 작용제를 포함하는 조성물, 및 사용 방법
KR102619197B1 (ko) 2017-01-23 2024-01-03 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
US11180756B2 (en) 2017-03-09 2021-11-23 Ionis Pharmaceuticals Morpholino modified oligomeric compounds
WO2018183969A1 (en) 2017-03-30 2018-10-04 California Institute Of Technology Barcoded rapid assay platform for efficient analysis of candidate molecules and methods of making and using the platform
WO2018195165A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
JP7801082B2 (ja) 2017-04-20 2026-01-16 シンセナ アーゲー トリシクロdnaヌクレオシドを含む改変オリゴマー化合物およびその使用
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
JP2020530442A (ja) 2017-07-10 2020-10-22 ジェンザイム・コーポレーション 血友病を有する対象の出血事象を処置するための方法および組成物
WO2019014262A1 (en) 2017-07-11 2019-01-17 The Scripps Research Institute INCORPORATION OF NON-NATURAL NUCLEOTIDES AND METHODS OF IN VIVO USE THEREOF
MA49578A (fr) 2017-07-11 2021-04-07 Synthorx Inc Incorporation de nucléotides non naturels et procédés associés
JP7277432B2 (ja) 2017-07-13 2023-05-19 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 乳酸脱水素酵素A(LDHA)iRNA組成物及びその使用方法
AU2018314159A1 (en) 2017-07-13 2020-01-30 Northwestern University General and direct method for preparing oligonucleotide-functionalized metal-organic framework nanoparticles
IL322312A (en) 2017-08-03 2025-09-01 Synthorx Inc Cytokine conjugates for the treatment of infectious diseases and cell proliferation
CA3071033A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
US11999953B2 (en) 2017-09-13 2024-06-04 The Children's Medical Center Corporation Compositions and methods for treating transposon associated diseases
US11806360B2 (en) 2017-09-19 2023-11-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
AU2018360697A1 (en) 2017-11-01 2020-05-14 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
EP3710587A1 (en) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
EP3714053B1 (en) 2017-11-22 2025-12-31 The University of Chicago CHEMICALLY DEPENDENT PROBE EVALUATION OF PROTEIN ACTIVITY AND ITS USES
EA202091520A1 (ru) 2017-12-18 2020-10-05 Элнилэм Фармасьютикалз, Инк. Композиции на основе irna против бокса-1 высокомобильной группы (hmgb1) и способы их применения
US11459564B2 (en) 2017-12-21 2022-10-04 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
EP3732187A4 (en) 2017-12-29 2021-11-10 The Scripps Research Institute INNATURAL BASE PAIR COMPOSITIONS AND METHODS OF USE
WO2019140452A1 (en) 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
CA3091857A1 (en) 2018-02-26 2019-08-29 Synthorx, Inc. Il-15 conjugates and uses thereof
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
EP3768694A4 (en) 2018-03-22 2021-12-29 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
US12473598B2 (en) 2018-03-28 2025-11-18 Board Of Regents, The University Of Texas System Identification of epigenetic alterations in DNA isolated from exosomes
US20210155959A1 (en) 2018-04-06 2021-05-27 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
KR20200141481A (ko) 2018-04-11 2020-12-18 아이오니스 파마수티컬즈, 인코포레이티드 Ezh2 발현의 조절제
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
BR112020020957B1 (pt) 2018-05-09 2022-05-10 Ionis Pharmaceuticals, Inc Compostos oligoméricos, população e composição farmacêutica dos mesmos e seus usos
CA3098144A1 (en) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
WO2019241648A1 (en) 2018-06-14 2019-12-19 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing stmn2 expression
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
WO2020018558A1 (en) 2018-07-17 2020-01-23 Aronora, Inc. Methods for safely reducing thrombopoietin
EP3826645A4 (en) 2018-07-25 2023-05-17 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING ATXN2 EXPRESSION
CA3106701A1 (en) 2018-08-13 2020-02-20 Alnylam Pharmaceuticals, Inc. Hepatitis b virus (hbv) dsrna agent compositions and methods of use thereof
TW202020157A (zh) 2018-08-16 2020-06-01 美商艾爾妮蘭製藥公司 用於抑制lect2基因表現之組合物及方法
CN112912422A (zh) 2018-09-14 2021-06-04 西北大学 用dna编程蛋白质聚合
WO2020060986A1 (en) 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
US12258620B2 (en) 2018-09-25 2025-03-25 Co-Diagnostics, Inc. Allele-specific design of cooperative primers for improved nucleic acid variant genotyping
AU2019350765A1 (en) 2018-09-28 2021-05-27 Alnylam Pharmaceuticals, Inc. Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated ocular diseases
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
MA54195A (fr) 2018-11-08 2022-02-16 Synthorx Inc Conjugués d'interleukine 10 et leurs utilisations
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
WO2020102630A1 (en) 2018-11-15 2020-05-22 Ionis Pharmaceuticals, Inc. Modulators of irf5 expression
BR112021007476A2 (pt) 2018-11-21 2021-11-03 Ionis Pharmaceuticals Inc Compostos e métodos para reduzir a expressão de príon
EP3894559A4 (en) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY
US20210332495A1 (en) 2018-12-06 2021-10-28 Northwestern University Protein Crystal Engineering Through DNA Hybridization Interactions
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ, インコーポレイテッド Kcnt1関連障害の治療のための組成物及び方法
LT3897672T (lt) 2018-12-20 2023-11-10 Vir Biotechnology, Inc. Kombinuota hbv terapija
BR112021013956A2 (pt) 2019-01-16 2021-09-21 Genzyme Corporation Composições de irna de serpinc1 e métodos de uso das mesmas
EP3917540A1 (en) 2019-01-31 2021-12-08 Ionis Pharmaceuticals, Inc. Modulators of yap1 expression
CA3127689A1 (en) 2019-02-06 2020-08-13 Synthorx, Inc. Il-2 conjugates and methods of use thereof
CA3131700A1 (en) 2019-02-27 2020-09-03 Ionis Pharmaceuticals, Inc. Modulators of malat1 expression
WO2020191177A1 (en) 2019-03-21 2020-09-24 Sudhir Agrawal Antisense oligonucleotides for allele specificity
JP7667074B2 (ja) 2019-03-29 2025-04-22 田辺三菱製薬株式会社 Dux4の発現を調節するための化合物、方法及び医薬組成物
HUE071494T2 (hu) 2019-03-29 2025-09-28 Ionis Pharmaceuticals Inc Vegyületek és eljárások az UBE3A-ATS modulálására
AU2020276243B2 (en) 2019-05-13 2025-12-18 Vir Biotechnology, Inc. Compositions and methods for treating hepatitis B virus (HBV) infection
EP3969052A1 (en) 2019-05-17 2022-03-23 Alnylam Pharmaceuticals Inc. Oral delivery of oligonucleotides
TW202544247A (zh) 2019-06-14 2025-11-16 美商史基普研究協會 於半合成生物體中複製、轉錄及轉譯之試劑及方法
AU2020295796A1 (en) 2019-06-18 2022-02-10 Janssen Sciences Ireland Unlimited Company Combination of hepatitis B virus (HBV) vaccines and HBV-targeting rnai
US20220305107A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi
EP3956450B1 (en) 2019-07-26 2025-08-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
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
CN114555621B (zh) 2019-08-15 2025-10-28 Ionis制药公司 键修饰的寡聚化合物及其用途
KR20220047598A (ko) 2019-08-15 2022-04-18 신톡스, 인크. Il-2 콘쥬게이트를 사용한 면역 종양학 병용 요법
CA3148135A1 (en) 2019-08-23 2021-03-04 Carolina E. CAFFARO Il-15 conjugates and uses thereof
JP7805286B2 (ja) 2019-09-03 2026-01-23 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 Lect2遺伝子の発現を阻害するための組成物および方法
CN114746122A (zh) 2019-09-10 2022-07-12 新索思股份有限公司 Il-2缀合物和治疗自身免疫性疾病的使用方法
US12319711B2 (en) 2019-09-20 2025-06-03 Northwestern University Spherical nucleic acids with tailored and active protein coronae
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
WO2021076828A1 (en) 2019-10-18 2021-04-22 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
AU2020369515A1 (en) 2019-10-22 2022-04-21 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
US12378560B2 (en) 2019-10-29 2025-08-05 Northwestern University Sequence multiplicity within spherical nucleic acids
AR120341A1 (es) 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
TWI891672B (zh) 2019-11-04 2025-08-01 美商欣爍克斯公司 介白素10接合物及其用途
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
CA3160329A1 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
TWI897902B (zh) 2019-12-13 2025-09-21 美商阿尼拉製藥公司 人類染色體9開讀框72 (C9ORF72) iRNA劑組成物及其使用方法
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類PATATIN磷脂酶結構域3(PNPLA3)iRNA組成物及其使用方法
US12503695B2 (en) 2020-01-27 2025-12-23 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services RAB13 and NET1 antisense oligonucleotides to treat metastatic cancer
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)
IL295445A (en) 2020-02-10 2022-10-01 Alnylam Pharmaceuticals Inc Preparations and methods for silencing vegf-a expression
KR20220143106A (ko) 2020-02-18 2022-10-24 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 C3 (APOC3) iRNA 조성물 및 이의 사용 방법
IL295605A (en) 2020-02-28 2022-10-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating smn2
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
IL296109A (en) 2020-03-06 2022-11-01 Alnylam Pharmaceuticals Inc Ketohexokinase (khk) irna compositions and methods of use thereof
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
JP2023519274A (ja) 2020-03-26 2023-05-10 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 コロナウイルスiRNA組成物およびその使用方法
WO2021202443A2 (en) 2020-03-30 2021-10-07 Alnylam Pharmaceucticals, Inc. Compositions and methods for silencing dnajc15 gene expression
EP4127134A4 (en) 2020-04-01 2024-07-31 Alnylam Pharmaceuticals, Inc. COMPOSITIONS OF ALPHA-2A ADRENERGIC RECEPTOR (ADRA2A) ARNI AGENTS AND METHODS OF USE THEREOF
EP4133078A1 (en) 2020-04-06 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
KR20230008078A (ko) 2020-04-07 2023-01-13 알닐람 파마슈티칼스 인코포레이티드 Scn9a 발현을 사일런싱하기 위한 조성물 및 방법
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
BR112022021813A2 (pt) 2020-04-27 2023-01-17 Alnylam Pharmaceuticals Inc Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas
WO2021222549A1 (en) 2020-04-30 2021-11-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
BR112022021333A2 (pt) 2020-05-01 2022-12-13 Ionis Pharmaceuticals Inc Compostos e métodos para modular atxn1
EP4151237A4 (en) 2020-05-12 2024-08-14 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for regulating expression of ataxin 3
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
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)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
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)
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)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
JP2023530234A (ja) 2020-06-05 2023-07-14 ザ・ブロード・インスティテュート・インコーポレイテッド 新生物を治療するための組成物および方法
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
CN116075592A (zh) 2020-06-09 2023-05-05 阿尔尼拉姆医药品有限公司 用于沉默gpam(线粒体甘油-3-磷酸酰基转移酶1)表达的sirna组合物和方法
AU2021292296A1 (en) 2020-06-18 2023-01-19 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof
AR122722A1 (es) 2020-06-24 2022-09-28 Vir Biotechnology Inc Anticuerpos que neutralizan el virus de la hepatitis b y sus usos
MX2022016254A (es) 2020-06-25 2023-04-11 Synthorx Inc Terapia de combinación de inmunooncología con conjugados de il-2 y anticuerpos anti-egfr.
MX2022016338A (es) 2020-06-29 2023-01-24 Ionis Pharmaceuticals Inc Compuestos y metodos para modular la plp1.
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
MX2023001222A (es) 2020-07-28 2023-04-26 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de app.
MX2023001486A (es) 2020-08-07 2023-03-27 Ionis Pharmaceuticals Inc Compuestos y metodos para modular scn2a.
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
EP3978608A1 (en) 2020-10-05 2022-04-06 SQY Therapeutics Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
EP4225917A1 (en) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
TW202228785A (zh) 2020-10-09 2022-08-01 美商欣爍克斯公司 使用il-2接合物及帕博利珠單抗(pembrolizumab)之免疫腫瘤學組合療法
AU2021356610A1 (en) 2020-10-09 2023-06-15 Synthorx, Inc. Immuno oncology therapies with il-2 conjugates
CA3198823A1 (en) 2020-10-21 2022-04-28 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
CA3196205A1 (en) 2020-10-23 2022-04-28 Floyd E. Romesberg Reverse transcription of polynucleotides comprising unnatural nucleotides
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc Coagulation factor iRNA compositions (F5) and methods of using them
EP4488371A3 (en) 2020-11-18 2025-04-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
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
CN112480197B (zh) * 2020-12-02 2022-03-08 新乡拓新药业股份有限公司 一种合成胞嘧啶核苷的方法
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
IL303800A (en) 2020-12-18 2023-08-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating factor xii
US20240175020A1 (en) 2020-12-23 2024-05-30 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
WO2022147223A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
AU2021411579A1 (en) 2020-12-31 2023-07-13 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2022150260A1 (en) 2021-01-05 2022-07-14 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4291243A1 (en) 2021-02-12 2023-12-20 Synthorx, Inc. Lung cancer combination therapy with il-2 conjugates and an anti-pd-1 antibody or antigen-binding fragment thereof
TW202305131A (zh) 2021-02-12 2023-02-01 美商艾拉倫製藥股份有限公司 用於治療或預防超氧歧化酶1-(SOD1-)相關的神經退化疾病的超氧歧化酶1(SOD1)iRNA組成物及其使用方法
TW202245843A (zh) 2021-02-12 2022-12-01 美商欣爍克斯公司 Il-2接合物及西米普利單抗(cemiplimab)之皮膚癌組合療法
JP2024509783A (ja) 2021-02-25 2024-03-05 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 プリオンタンパク質(prnp)irna組成物およびその使用方法
EP4298218A1 (en) 2021-02-26 2024-01-03 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
IL305442A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
EP4304640A1 (en) 2021-03-12 2024-01-17 Northwestern University Antiviral vaccines using spherical nucleic acids
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
IL307298A (en) 2021-03-31 2023-11-01 Entrada Therapeutics Inc Cell-penetrating circular peptides
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
CA3216106A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
WO2022235537A1 (en) 2021-05-03 2022-11-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
WO2022240721A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
KR20240012425A (ko) 2021-05-10 2024-01-29 엔트라다 테라퓨틱스, 인크. 세포내 치료제를 위한 조성물 및 방법
US20250051780A1 (en) 2021-05-10 2025-02-13 Entrada Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
US20240263177A1 (en) 2021-05-20 2024-08-08 Korro Bio, Inc. Methods and Compositions for Adar-Mediated Editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
TW202313117A (zh) 2021-06-03 2023-04-01 美商欣爍克斯公司 包含il-2接合物及西妥昔單抗(cetuximab)之頭頸癌組合療法
EP4347822A2 (en) 2021-06-04 2024-04-10 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
BR112023026050A2 (pt) 2021-06-18 2024-03-05 Ionis Pharmaceuticals Inc Compostos e métodos para reduzir expressão de ifnar1
KR20240038967A (ko) 2021-06-23 2024-03-26 엔트라다 테라퓨틱스, 인크. Cug 반복을 표적화하기 위한 안티센스 화합물 및 방법
EP4363574A1 (en) 2021-06-29 2024-05-08 Korro Bio, Inc. Methods and compositions for adar-mediated editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
WO2023003805A1 (en) 2021-07-19 2023-01-26 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder
MX2024000981A (es) 2021-07-21 2024-02-12 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de gen diana asociado con trastorno metabolico y sus metodos de uso.
AU2022316139A1 (en) 2021-07-23 2024-01-18 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
JP2024529437A (ja) 2021-07-29 2024-08-06 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 3-ヒドロキシ-3-メチルグリタル-COAレダクターゼ(HMGCR)iRNA組成物およびその使用方法
CN117795074A (zh) 2021-08-03 2024-03-29 阿尔尼拉姆医药品有限公司 转甲状腺素蛋白(TTR)iRNA组合物和其使用方法
PE20241132A1 (es) 2021-08-04 2024-05-24 Alnylam Pharmaceuticals Inc Composiciones de arni y metodos para silenciar el angiotensinogeno (agt)
JP2024534766A (ja) 2021-08-13 2024-09-26 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 第XII因子(F12)iRNA組成物およびその使用方法
EP4395790A2 (en) 2021-08-31 2024-07-10 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
KR20240082344A (ko) 2021-09-01 2024-06-10 엔트라다 테라퓨틱스, 인크. 뒤센 근이영양증에서 엑손 44를 스키핑하기 위한 화합물 및 방법
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
CA3232420A1 (en) 2021-09-20 2023-03-23 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
MX2024003519A (es) 2021-09-24 2024-04-01 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) de proteina tau asociada a microtubulos (mapt) y sus metodos de uso.
WO2023056440A1 (en) 2021-10-01 2023-04-06 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof
KR20240101590A9 (ko) 2021-10-15 2025-12-10 알닐람 파마슈티칼스 인코포레이티드 간외 전달 irna 조성물 및 이를 이용하는 방법
US20250352667A1 (en) 2021-10-22 2025-11-20 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
EP4423273A1 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2023092060A1 (en) 2021-11-18 2023-05-25 Cornell University Microrna-dependent mrna switches for tissue-specific mrna-based therapies
WO2023122573A1 (en) 2021-12-20 2023-06-29 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
JP2025516680A (ja) 2022-05-13 2025-05-30 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 一本鎖ループオリゴヌクレオチド
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
JP2025532593A (ja) 2022-09-15 2025-10-01 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 17b-ヒドロキシステロイドデヒドロゲナーゼ13型(hsd17b13)irna組成物およびその使用方法
WO2024064858A2 (en) 2022-09-23 2024-03-28 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing mecp2 expression
JP2025532985A (ja) 2022-09-30 2025-10-03 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 修飾二本鎖rna剤
WO2024136899A1 (en) 2022-12-21 2024-06-27 Synthorx, Inc. Cancer therapy with il-2 conjugates and chimeric antigen receptor therapies
CN120813691A (zh) 2023-02-09 2025-10-17 阿尔尼拉姆医药品有限公司 Reversir分子及其使用方法
EP4665865A1 (en) 2023-02-17 2025-12-24 Anjarium Biosciences AG Methods of making dna molecules and compositions and uses thereof
WO2024196937A1 (en) 2023-03-20 2024-09-26 Synthorx, Inc. Cancer therapy with il-2 peg conjugates
WO2024216155A1 (en) 2023-04-12 2024-10-17 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
WO2024220930A2 (en) 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024226499A1 (en) 2023-04-24 2024-10-31 The Broad Institute, Inc. Compositions and methods for modifying fertility
WO2024238396A1 (en) 2023-05-12 2024-11-21 Adarx Pharmaceuticals, Inc. Nmda ligand conjugated compounds and uses thereof
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
CN121335980A (zh) 2023-05-26 2026-01-13 阿达尔克斯制药有限公司 Sod1调节组合物及其使用方法
AU2024313301A1 (en) 2023-06-20 2025-12-11 Adarx Pharmaceuticals, Inc. Lrrk2-modulating compositions and methods of use thereof
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025024334A1 (en) 2023-07-21 2025-01-30 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025072672A2 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
WO2025072699A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Aminoglycosides for delivery of agents to the kidney
WO2025072713A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Polymyxins for delivery of agents to the kidney
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
AU2024369609A1 (en) 2023-10-31 2025-12-11 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
WO2025178854A2 (en) 2024-02-19 2025-08-28 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting cideb and related methods
WO2025199231A2 (en) 2024-03-20 2025-09-25 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
WO2025207517A2 (en) 2024-03-25 2025-10-02 Synthorx, Inc. Synthetic trna synthetases and cells comprising synthetic molecules for production of polypeptides
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2025259743A1 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dual conjugate compounds for extrahepatic delivery
WO2025259747A2 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof
WO2026006436A1 (en) 2024-06-25 2026-01-02 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of tar dna binding protein 43 kda (tdp43)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4806463A (en) * 1986-05-23 1989-02-21 Worcester Foundation For Experimental Biology Inhibition of HTLV-III by exogenous oligonucleotides
EP0266099A3 (en) * 1986-10-28 1990-09-19 The Johns Hopkins University Oligonucleoside alkyl or arylphosphonate derivatives capable of crosslinking with or cleaving nucleic acids
IL84414A0 (en) * 1986-11-14 1988-04-29 Ciba Geigy Ag N9-cyclopentyl-substituted adenine derivatives
IN166499B (enExample) * 1987-02-12 1990-05-19 Takeda Chemical Industries Ltd
AU636573B2 (en) * 1988-02-26 1993-05-06 Worcester Foundation For Biomedical Research, Inc. Inhibition of htlv-iii by exogenous oligonucleotides
EP0942000B1 (en) * 1989-10-24 2004-06-23 Isis Pharmaceuticals, Inc. 2'-Modified oligonucleotides

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US5461152A (en) 1995-10-24
US5700920A (en) 1997-12-23
CA2099712A1 (en) 1994-01-02
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