IL161827A0 - A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby - Google Patents

A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby

Info

Publication number
IL161827A0
IL161827A0 IL16182702A IL16182702A IL161827A0 IL 161827 A0 IL161827 A0 IL 161827A0 IL 16182702 A IL16182702 A IL 16182702A IL 16182702 A IL16182702 A IL 16182702A IL 161827 A0 IL161827 A0 IL 161827A0
Authority
IL
Israel
Prior art keywords
sequences
detecting
isolating
identified
aav
Prior art date
Application number
IL16182702A
Other languages
English (en)
Original Assignee
Univ Pennsylvania
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=27502639&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=IL161827(A0) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of IL161827A0 publication Critical patent/IL161827A0/xx

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/70—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
    • C12Q1/701—Specific hybridization probes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/06—Antipsoriatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6816—Hybridisation assays characterised by the detection means
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14161—Methods of inactivation or attenuation
    • C12N2750/14162—Methods of inactivation or attenuation by genetic engineering
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/90—Vector systems having a special element relevant for transcription from vertebrates avian
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Neurology (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Cell Biology (AREA)
  • Endocrinology (AREA)
IL16182702A 2001-11-13 2002-11-12 A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby IL161827A0 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US35060701P 2001-11-13 2001-11-13
US34111701P 2001-12-17 2001-12-17
US37706602P 2002-05-01 2002-05-01
US38667502P 2002-06-05 2002-06-05
PCT/US2002/033629 WO2003042397A2 (fr) 2001-11-13 2002-11-12 Methode de detection et/ou d'identification de sequences de virus associes aux adenovirus (aav) et d'isolation de nouvelles sequences ainsi identifiees

Publications (1)

Publication Number Publication Date
IL161827A0 true IL161827A0 (en) 2005-11-20

Family

ID=27502639

Family Applications (11)

Application Number Title Priority Date Filing Date
IL16182702A IL161827A0 (en) 2001-11-13 2002-11-12 A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby
IL270065A IL270065B2 (en) 2001-11-13 2002-11-12 Recombinant AAV viruses, method of their creation and uses
IL161827A IL161827A (en) 2001-11-13 2004-05-06 Method for tracking and / or identifying sequences of a virus belonging to the adenoid AAV type) and isolating new sequences identified as such
IL193525A IL193525A (en) 2001-11-13 2008-08-18 Serological type 7 adenoid virus sequences and recombinant viruses containing them
IL213810A IL213810A (en) 2001-11-13 2011-06-28 Virus sequences belonging to adenoid type (aav) 8 and recombinant viruses containing them
IL221602A IL221602A (en) 2001-11-13 2012-08-23 Virus sequences belonging to the rh10 adenoid type and recombinant viruses containing them
IL227866A IL227866A (en) 2001-11-13 2013-08-08 A method for tracking and / or identifying adenoid (aav) virus sequences and isolating new sequences identified as such
IL233391A IL233391A (en) 2001-11-13 2014-06-26 Capsid-containing aav recombinant virus containing aav protein cg.5 vp3, a minigan containing reverse aav tip and a heterologous gene
IL234063A IL234063A (en) 2001-11-13 2014-08-11 Adenovirus containing capsid aavrh.34 and a heterologous gene, its preparation and preparations containing it
IL248724A IL248724B (en) 2001-11-13 2016-11-03 Recombinant adeno-associated viruses (aavs), a method for their creation and uses
IL25854518A IL258545B (en) 2001-11-13 2018-04-08 Recombinant aav viruses, a method for their creation and uses

Family Applications After (10)

Application Number Title Priority Date Filing Date
IL270065A IL270065B2 (en) 2001-11-13 2002-11-12 Recombinant AAV viruses, method of their creation and uses
IL161827A IL161827A (en) 2001-11-13 2004-05-06 Method for tracking and / or identifying sequences of a virus belonging to the adenoid AAV type) and isolating new sequences identified as such
IL193525A IL193525A (en) 2001-11-13 2008-08-18 Serological type 7 adenoid virus sequences and recombinant viruses containing them
IL213810A IL213810A (en) 2001-11-13 2011-06-28 Virus sequences belonging to adenoid type (aav) 8 and recombinant viruses containing them
IL221602A IL221602A (en) 2001-11-13 2012-08-23 Virus sequences belonging to the rh10 adenoid type and recombinant viruses containing them
IL227866A IL227866A (en) 2001-11-13 2013-08-08 A method for tracking and / or identifying adenoid (aav) virus sequences and isolating new sequences identified as such
IL233391A IL233391A (en) 2001-11-13 2014-06-26 Capsid-containing aav recombinant virus containing aav protein cg.5 vp3, a minigan containing reverse aav tip and a heterologous gene
IL234063A IL234063A (en) 2001-11-13 2014-08-11 Adenovirus containing capsid aavrh.34 and a heterologous gene, its preparation and preparations containing it
IL248724A IL248724B (en) 2001-11-13 2016-11-03 Recombinant adeno-associated viruses (aavs), a method for their creation and uses
IL25854518A IL258545B (en) 2001-11-13 2018-04-08 Recombinant aav viruses, a method for their creation and uses

Country Status (23)

Country Link
US (15) US20030138772A1 (fr)
EP (4) EP2338900B1 (fr)
JP (6) JP3958191B2 (fr)
KR (2) KR101014207B1 (fr)
CN (6) CN102181480B (fr)
AT (2) ATE520707T1 (fr)
AU (1) AU2002361573B2 (fr)
BR (3) BR122016004546B8 (fr)
CA (8) CA2756866C (fr)
DE (1) DE60209193T2 (fr)
DK (1) DK1310571T3 (fr)
ES (3) ES2455126T3 (fr)
HK (2) HK1056198A1 (fr)
HU (2) HU229379B1 (fr)
IL (11) IL161827A0 (fr)
MX (3) MX346493B (fr)
NO (4) NO334379B1 (fr)
NZ (7) NZ591012A (fr)
PH (2) PH12014501487A1 (fr)
PL (4) PL222683B1 (fr)
SG (5) SG10202108118RA (fr)
WO (1) WO2003042397A2 (fr)
ZA (1) ZA200403360B (fr)

Families Citing this family (521)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
CA2745131C (fr) 1998-05-28 2016-08-09 John A. Chiorini Vecteurs d'aav5 et leurs utilisation
US7226739B2 (en) 2001-03-02 2007-06-05 Isis Pharmaceuticals, Inc Methods for rapid detection and identification of bioagents in epidemiological and forensic investigations
US7718354B2 (en) 2001-03-02 2010-05-18 Ibis Biosciences, Inc. Methods for rapid identification of pathogens in humans and animals
US20030027135A1 (en) 2001-03-02 2003-02-06 Ecker David J. Method for rapid detection and identification of bioagents
US7666588B2 (en) 2001-03-02 2010-02-23 Ibis Biosciences, Inc. Methods for rapid forensic analysis of mitochondrial DNA and characterization of mitochondrial DNA heteroplasmy
US20040121311A1 (en) 2002-12-06 2004-06-24 Ecker David J. Methods for rapid detection and identification of bioagents in livestock
US7217510B2 (en) 2001-06-26 2007-05-15 Isis Pharmaceuticals, Inc. Methods for providing bacterial bioagent characterizing information
US8073627B2 (en) 2001-06-26 2011-12-06 Ibis Biosciences, Inc. System for indentification of pathogens
ATE520707T1 (de) * 2001-11-13 2011-09-15 Univ Pennsylvania Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
EP2359869B1 (fr) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Séquences de virus adéno-associés de sérotype 8 , vecteurs les contenant et leurs utilisations
ES2526341T3 (es) * 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
CA2426283C (fr) 2002-04-29 2006-06-27 The Trustees Of The University Of Pennsylvania Methode d'isolement et d'amplification directs de virus integres dans l'adn cellulaire de tissus
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US7220577B2 (en) * 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. Modified AAV
JP2006516193A (ja) 2002-12-06 2006-06-29 アイシス・ファーマシューティカルス・インコーポレーテッド ヒトおよび動物における病原体の迅速な同定方法
US8046171B2 (en) 2003-04-18 2011-10-25 Ibis Biosciences, Inc. Methods and apparatus for genetic evaluation
US8057993B2 (en) 2003-04-26 2011-11-15 Ibis Biosciences, Inc. Methods for identification of coronaviruses
US8158354B2 (en) 2003-05-13 2012-04-17 Ibis Biosciences, Inc. Methods for rapid purification of nucleic acids for subsequent analysis by mass spectrometry by solution capture
US7964343B2 (en) 2003-05-13 2011-06-21 Ibis Biosciences, Inc. Method for rapid purification of nucleic acids for subsequent analysis by mass spectrometry by solution capture
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
ES2862206T3 (es) 2003-06-19 2021-10-07 Genzyme Corp Viriones de AAV con inmunorreactividad disminuida y sus usos
US9233131B2 (en) * 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
NZ545656A (en) * 2003-09-01 2009-02-28 Amc Amsterdam AAV vectors for in vivo gene therapy of rheumatoid arthritis
AU2010201278B2 (en) * 2003-09-01 2012-11-15 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
US8097416B2 (en) 2003-09-11 2012-01-17 Ibis Biosciences, Inc. Methods for identification of sepsis-causing bacteria
US8546082B2 (en) 2003-09-11 2013-10-01 Ibis Biosciences, Inc. Methods for identification of sepsis-causing bacteria
US8288523B2 (en) 2003-09-11 2012-10-16 Ibis Biosciences, Inc. Compositions for use in identification of bacteria
EP2298926A1 (fr) * 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Clades (sous-types) et séquences de Virus Adeno-Associé (AAV), vecteurs les contenant et leurs utilistation
AU2011250849B2 (en) * 2003-09-30 2013-09-12 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
WO2005056807A2 (fr) * 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Vecteur viral associe aux adenovirus bovins et leurs utilisations
US8163895B2 (en) 2003-12-05 2012-04-24 Ibis Biosciences, Inc. Compositions for use in identification of orthopoxviruses
US7666592B2 (en) 2004-02-18 2010-02-23 Ibis Biosciences, Inc. Methods for concurrent identification and quantification of an unknown bioagent
US8119336B2 (en) 2004-03-03 2012-02-21 Ibis Biosciences, Inc. Compositions for use in identification of alphaviruses
DE602005026269D1 (de) 2004-04-28 2011-03-24 Univ Pennsylvania Sequenzielle freisetzung immunogener moleküle über adenoviren und adeno-assoziierte viren vermittelte abgaben
ES2442225T3 (es) 2004-04-28 2014-02-10 The Trustees Of The University Of Pennsylvania Régimen de inmunización con cebado de adenovirus suprimido en E4 y potenciación de adenovirus suprimido en E1
CA2567839C (fr) 2004-05-24 2011-06-28 Isis Pharmaceuticals, Inc. Spectrometrie de masse a filtration ionique selective par seuillage numerique
US20050266411A1 (en) 2004-05-25 2005-12-01 Hofstadler Steven A Methods for rapid forensic analysis of mitochondrial DNA
US7811753B2 (en) 2004-07-14 2010-10-12 Ibis Biosciences, Inc. Methods for repairing degraded DNA
EP1771571A2 (fr) * 2004-07-30 2007-04-11 Targeted Genetics Corporation Procedes de vaccination a base d'aav recombine
US7309589B2 (en) * 2004-08-20 2007-12-18 Vironix Llc Sensitive detection of bacteria by improved nested polymerase chain reaction targeting the 16S ribosomal RNA gene and identification of bacterial species by amplicon sequencing
CA2580221A1 (fr) * 2004-10-05 2006-04-13 Merz Pharma Gmbh & Co. Kgaa Nouvelles propenones cycliques et acycliques destinees au traitement des troubles du snc
EP1869180B1 (fr) 2005-03-03 2013-02-20 Ibis Biosciences, Inc. Compositions utilisées pour identifier des virus polyoma
US8084207B2 (en) 2005-03-03 2011-12-27 Ibis Bioscience, Inc. Compositions for use in identification of papillomavirus
EP3409296A1 (fr) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Procédé d'amélioration de la fonction d'un vecteur aav
WO2006119432A2 (fr) * 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, clonage, et caracterisation de nouveaux serotypes de virus adeno-associes (avv)
US8026084B2 (en) 2005-07-21 2011-09-27 Ibis Biosciences, Inc. Methods for rapid identification and quantitation of nucleic acid variants
EP1957678B1 (fr) * 2005-11-28 2012-06-13 Ibis Biosciences, Inc. Compositions destinees a etre utilisees pour l'identification de virus contaminants adventices
WO2007120542A2 (fr) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Bibliothèque de capsides aav et protéines de capsides aav
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
PL2044199T3 (pl) * 2006-07-25 2013-04-30 Celladon Corp Przedłużony, podawany z prądem krwi, nasierdziowy, wieńcowy wlew wektorów wirusowych na bazie wirusów towarzyszących adenowirusom, zawierający SERCA2a do terapii genowej
WO2008143627A2 (fr) 2006-09-14 2008-11-27 Ibis Biosciences, Inc. Procédé d'amplification ciblée de génome entier pour l'identification d'agents pathogènes
EP2126132B1 (fr) 2007-02-23 2013-03-20 Ibis Biosciences, Inc. Procédé d'analyse d'adn médico-légale rapide
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
US9598724B2 (en) 2007-06-01 2017-03-21 Ibis Biosciences, Inc. Methods and compositions for multiple displacement amplification of nucleic acids
EP2058401A1 (fr) * 2007-10-05 2009-05-13 Genethon Fourniture généralisée de gènes à des motoneurones utilisant l'injection périphérique de vecteurs AAV
MX2010012592A (es) 2008-05-20 2011-05-05 Eos Neuroscience Inc Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas.
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010033627A2 (fr) 2008-09-16 2010-03-25 Ibis Biosciences, Inc. Unités de traitement d'échantillons, systèmes et procédés associés
EP2349549B1 (fr) 2008-09-16 2012-07-18 Ibis Biosciences, Inc. Cartouches de mélange, postes de mélange et kits, et système associé
EP2344893B1 (fr) 2008-09-16 2014-10-15 Ibis Biosciences, Inc. Systèmes et procédés de manipulation de microplaques
EP2396803A4 (fr) 2009-02-12 2016-10-26 Ibis Biosciences Inc Ensembles sonde d'ionisation
EP2425000B1 (fr) 2009-04-30 2019-02-20 The Trustees Of The University Of Pennsylvania Compositions pour cibler des cellules des voies respiratoires conductrices comprenant des constructions de virus adéno-associé
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2010143761A1 (fr) * 2009-06-12 2010-12-16 (주)바이오니아 Procédé de détection rapide de microorganismes infectieux dans un échantillon inconnu
WO2011008972A1 (fr) 2009-07-17 2011-01-20 Ibis Biosciences, Inc. Systèmes pour l'identification d'un bioagent
US8950604B2 (en) 2009-07-17 2015-02-10 Ibis Biosciences, Inc. Lift and mount apparatus
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
WO2011047307A1 (fr) 2009-10-15 2011-04-21 Ibis Biosciences, Inc. Amplification par déplacement multiple
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
SG10201908848RA (en) 2010-03-29 2019-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
CA3066596A1 (fr) 2010-04-23 2011-10-27 University Of Massachusetts Vecteurs aav ciblant le systeme nerveux central et leurs procedes d'utilisation
CA2833912C (fr) 2010-04-23 2021-09-21 University Of Massachusetts Traitement a base d'aav pour des troubles lies au cholesterol
CA2833905C (fr) 2010-04-23 2019-09-10 University Of Massachusetts Constructions d'expression a cistrons multiples
CA3128549A1 (fr) 2010-07-12 2012-01-19 Universitat Autonoma De Barcelona Composition de therapie genique destinee a etre utilisee dans le traitement du diabete
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP3699286A1 (fr) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Régimes et compositions pour l'immunisation passive médiée par aav contre des agents pathogènes en suspension dans l'air
WO2012145624A2 (fr) 2011-04-21 2012-10-26 University Of Massachusetts Compositions à base de raav et procédés pour traiter des déficiences en anti-trypsine alpha-1
KR20220056884A (ko) 2011-04-22 2022-05-06 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법
US9169299B2 (en) * 2011-08-24 2015-10-27 The Board Of Trustees Of The Leleand Stanford Junior University AAV capsid proteins for nucleic acid transfer
US20130136729A1 (en) * 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
US20150111955A1 (en) * 2012-02-17 2015-04-23 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
WO2013130393A1 (fr) * 2012-02-28 2013-09-06 Cornell University Expression persistante dirigée contre aav d'un gène d'anticorps anti-nicotine pour l'abandon du tabac
WO2013154744A1 (fr) * 2012-04-13 2013-10-17 Cornell University Mise au point d'un vaccin conjugué hautement efficace de deuxième génération à base de nicotine destiné à traiter la dépendance à la nicotine
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2692868A1 (fr) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Vecteurs viraux adéno-associés (AAV) utiles pour la transduction de tissu adipeux
EP2954051B1 (fr) 2013-02-08 2019-03-27 The Trustees Of The University Of Pennsylvania Capsides aav8 modifiées pour transfert génétique pour thérapies rétiniennes
RS61387B1 (sr) 2013-02-15 2021-02-26 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
US8957044B2 (en) 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
JP6591956B2 (ja) 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｍｐｓ１を治療するための組成物および方法
WO2015012924A2 (fr) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Cassettes d'expression préférentielle pour tissus modifiées par un codon, vecteurs les contenant, et utilisation
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
SG10201809075XA (en) 2013-07-22 2018-11-29 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
ES2838804T3 (es) 2014-01-31 2021-07-02 Univ Temple BAG3 como diana para el tratamiento de la insuficiencia cardíaca
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (fr) 2014-02-19 2015-08-27 University Of Massachusetts Vaa recombinants ayant des propriétés de transcytose utiles
CA2939950C (fr) * 2014-03-09 2023-08-22 The Trustees Of The University Of Pennsylvania Compositions utiles dans le traitement du deficit en ornithine transcarbamylase (otc)
SG10201810150UA (en) 2014-03-17 2018-12-28 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
WO2015143078A1 (fr) 2014-03-18 2015-09-24 University Of Massachusetts Compositions à base de virus adéno-associé recombinant raav et méthodes de traitement de la sclérose latérale amyotrophique
FI3628334T3 (fi) 2014-03-21 2023-09-15 Genzyme Corp Geenihoito verkkokalvon pigmenttirappeuma
EP2933335A1 (fr) 2014-04-18 2015-10-21 Genethon Procédé de traitement de neuropathies périphériques et sclérose latérale amyotrophique
CA2946392A1 (fr) 2014-04-25 2015-10-29 James M. Wilson Variants de ldlr et leur utilisation dans des compositions permettant de reduire les taux de cholesterol
US10780182B2 (en) 2014-04-25 2020-09-22 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
EP3134522B1 (fr) 2014-04-25 2021-10-06 University of Massachusetts Vecteurs de virus adéno-associés recombinants utiles pour réduire une immunité contre des produits transgéniques
WO2015175639A1 (fr) 2014-05-13 2015-11-19 The Trustees Of The University Of Pennsylvania Compositions comprenant un virus adéno-associé exprimant des constructions à double anticorps et leurs utilisations
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2015191508A1 (fr) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Capsides chimériques
EP2960336A1 (fr) 2014-06-27 2015-12-30 Genethon Traitement systémique efficace de pathologies dystrophiques musculaires
US9809588B2 (en) 2014-07-03 2017-11-07 Board Of Regents, The University Of Texas System GLS1 inhibitors for treating disease
WO2016044478A1 (fr) 2014-09-16 2016-03-24 Genzyme Corporation Vecteurs viraux adéno-associés pour le traitement du glaucome à myociline (myoc)
EP4012035A1 (fr) 2014-09-16 2022-06-15 Genzyme Corporation Vecteurs viraux adéno-associés pour le traitement du glaucome à myociline (myoc)
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3200830B1 (fr) 2014-10-03 2020-09-09 University of Massachusetts Vecteurs aav identifiés au moyen de banques à efficacité élevée
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
DK3218386T3 (da) 2014-11-14 2021-06-07 Voyager Therapeutics Inc Modulatorisk polynukleotid
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
EP3230441A4 (fr) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions et méthodes pour la production de scaav
ES2900973T3 (es) 2015-01-07 2022-03-21 UNIV AUTòNOMA DE BARCELONA Constructo genético de vector individual que comprende genes de insulina y glucoquinasa
EP3632923A1 (fr) 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Polynucléotides de ciblage du système nerveux central
IL296391B2 (en) 2015-01-20 2024-06-01 Genzyme Corp Analytical ultracentrifugation for characterization of recombinant viral particles
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
SG10201907399RA (en) 2015-02-10 2019-09-27 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
WO2016141078A1 (fr) 2015-03-02 2016-09-09 Avalanche Biotechnologies, Inc. Compositions et procédés d'administration intravitréenne de polynucléotides à des cônes rétiniens
CA2978917A1 (fr) 2015-03-10 2016-09-15 The Trustees Of Columbia University In The City Of New York Constructions de vecteur viral adeno-associe glut1 de recombinaison et procedes associes permettant de restaurer l'expression de glut1
WO2016154344A1 (fr) 2015-03-24 2016-09-29 The Regents Of The University Of California Variants de virus adéno-associé et leurs procédés d'utilisation
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CA3019315A1 (fr) 2015-04-23 2016-10-27 University Of Massachusetts Modulation de l'expression d'un transgene du vecteur aav
CA3021949C (fr) 2015-04-24 2023-10-17 University Of Massachusetts Constructions d'aav modifiees et utilisations de celles-ci
JP6851319B2 (ja) 2015-04-27 2021-03-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
MA48852A (fr) 2015-05-13 2020-04-01 Univ Pennsylvania Expression médiée par aav d'anticorps anti-grippaux et leurs procédés d'utilisation
WO2016186772A2 (fr) 2015-05-16 2016-11-24 Genzyme Corporation Édition génique de mutations introniques profondes
IL256517B2 (en) 2015-06-23 2024-06-01 Childrens Hospital Philadelphia Modified factor IX, and preparations, methods and uses for gene transfer to cells, organs and tissues
EP3325018A4 (fr) 2015-07-22 2019-04-24 Duke University Criblage à haut rendement d'une fonction d'élément de régulation à l'aide de technologies d'édition de l'épigénome
AU2016302335B2 (en) 2015-08-06 2022-08-04 The Trustees Of The University Of Pennsylvania GLP-1 and use thereof in compositions for treating metabolic diseases
CA2996001A1 (fr) 2015-08-25 2017-03-02 Duke University Compositions et procedes d'amelioration de la specificite dans l'ingenierie genomique a l'aide d'endonucleases guidees par arn
WO2017040524A1 (fr) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo pour le traitement d'animaux de compagnie
JP7261583B2 (ja) 2015-09-24 2023-04-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 補体媒介性疾患を処置するための組成物及び方法
CA2996420A1 (fr) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Procedes et compositions pour vecteurs viraux evitant les anticorps
US11273227B2 (en) 2015-10-09 2022-03-15 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating Stargardt's disease and other ocular disorders
US11970710B2 (en) 2015-10-13 2024-04-30 Duke University Genome engineering with Type I CRISPR systems in eukaryotic cells
WO2017070525A1 (fr) 2015-10-22 2017-04-27 University Of Massachusetts Procédés et compositions pour le traitement du déséquilibre métabolique dans une maladie neurodégénérative
WO2017070516A1 (fr) 2015-10-22 2017-04-27 University Of Massachusetts Vecteurs du sérotype de virus adéno-associés ciblant la prostate
WO2017075119A1 (fr) 2015-10-28 2017-05-04 The Trustees Of The Univeresity Of Pennsylvania Administration intrathécale de vecteurs viraux adéno-associés pour la thérapie génique
EP3368054A4 (fr) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. Expression régulable au moyen d'un virus adéno-associé (vaa)
WO2017072361A1 (fr) 2015-10-30 2017-05-04 Nbe-Therapeutics Ag Anticorps anti-ror1
CA3006569A1 (fr) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Dosages pour la detection d'anticorps neutralisants de vaa
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
WO2017100704A1 (fr) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif d'aavrh10
CN115957350A (zh) 2015-12-11 2023-04-14 宾夕法尼亚州大学信托人 用于治疗家族性高胆固醇血症的基因疗法
WO2017160360A2 (fr) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Méthode de purification évolutive pour virus adéno-associé 9 (aav9)
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
EP4215605A1 (fr) 2015-12-11 2023-07-26 The Trustees of The University of Pennsylvania Procédé de purification évolutif d'aav8
RU2762747C2 (ru) 2015-12-14 2021-12-22 Зе Трастис Оф Зе Юниверсити Оф Пенсильвания Генная терапия офтальмологических нарушений
WO2017106345A1 (fr) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Composition pour le traitement du syndrome de crigler-najjar
CA3008268A1 (fr) 2015-12-15 2017-06-22 Genzyme Corporation Vecteurs viraux adeno-associes pour traiter la mucolipidose de type ii
EP3405496B1 (fr) 2016-01-20 2023-10-25 University of Florida Research Foundation, Incorporated Compositions d'anticorps anti-ror1 et procédés associés
AU2017214378B2 (en) 2016-02-01 2023-05-04 Bioverativ Therapeutics Inc. Optimized Factor VIII genes
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
AU2017215211C1 (en) 2016-02-03 2023-11-16 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
EP4094780A3 (fr) 2016-02-12 2023-02-08 University of Massachusetts Thérapeutiques micro-arn anti-angiogéniques pour l'inhibition de la néovascularisation de la cornée
US10729789B2 (en) * 2016-03-01 2020-08-04 University Of Virginia Patent Foundation Compositions and methods for adeno-associated virus mediated gene expression in myofibroblast-like cells
EP3440210A4 (fr) 2016-04-05 2019-11-27 University of Massachusetts Compositions et procédés pour l'inhibition sélective de l'expression d'une protéine de type grainhead
US11446398B2 (en) 2016-04-11 2022-09-20 Obsidian Therapeutics, Inc. Regulated biocircuit systems
CA3019423A1 (fr) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Nouvelles capsides mutantes de aav8 et compositions les contenant
WO2017180936A1 (fr) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Composition pour le traitement de la dégénérescence maculaire liée a l'âge exsudative
KR20230125339A (ko) 2016-04-15 2023-08-29 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
WO2017180861A1 (fr) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsulvania Thérapie génique pour le traitement de l'hémophilie b
WO2017180857A1 (fr) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Thérapie génique pour le traitement de l'hémophilie a
WO2017181105A1 (fr) 2016-04-15 2017-10-19 University Of Massachusetts Méthodes et compositions pour le traitement de déséquilibres métaboliques
US11401527B2 (en) 2016-04-17 2022-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189959A1 (fr) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions pour le traitement de maladies
GB201608046D0 (en) * 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
WO2017197355A2 (fr) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Variantes de capsides de virus adéno-associé et leurs procédés d'utilisation
CN109831916B (zh) 2016-05-18 2023-07-21 沃雅戈治疗公司 治疗亨廷顿氏舞蹈病的组合物和方法
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc modulatory polynucleotides
US11158076B2 (en) 2016-06-08 2021-10-26 Sony Corporation Imaging control device and method, and vehicle
WO2017218852A1 (fr) 2016-06-15 2017-12-21 University Of Massachusetts Virus adéno-associés de recombinaison pour l'administration de molécules d'édition de gènes dans des cellules embryonnaires
KR20190096329A (ko) 2016-07-05 2019-08-19 유니버시티 오브 매사추세츠 녹내장에서 신경보호 요법으로서 sfasl의 aav2-매개된 유전자 전달
EP3481958A4 (fr) 2016-07-08 2019-12-25 The Trustees of The University of Pennsylvania Procédés et compositions de traitement de troubles et de maladies impliquant rdh12
WO2018022511A1 (fr) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprenant un variant de la lécithine-cholestérol-acyl-transférase et leurs utilisations
EP3491008A2 (fr) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Nouvelles protéines de capside de virus adéno-associés
CN116286986A (zh) 2016-07-29 2023-06-23 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
TN2019000047A1 (en) 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
EP3831281A1 (fr) 2016-08-30 2021-06-09 The Regents of The University of California Procédés de ciblage et d'administration biomédicaux, et dispositifs et systèmes pour leur mise en uvre
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
WO2018064624A1 (fr) * 2016-09-29 2018-04-05 University Of Florida Research Foundation, Incorporated Variantes aavrh.10 à capacités d'échappement d'anticorps hôtes et propriétés de ciblage de tissu modifiées
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
WO2018075798A1 (fr) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Capsides d'aav modifiées et leurs utilisations
US20190328846A1 (en) 2016-12-01 2019-10-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Pharmaceutical compositions for the treatment of retinal degenerative diseases
US11584931B2 (en) 2016-12-14 2023-02-21 The J. David Gladstone Institutes Methods and compositions for generating a deletion library and for identifying a defective interfering particle (DIP)
KR20190100318A (ko) * 2016-12-30 2019-08-28 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
CA3042467A1 (fr) 2017-01-20 2018-07-26 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Traitement de la maladie de krabbe par transplantation de sang de cordon ombilical (tsco) et expression accrue de la galactocerebrosidase (galc)
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SG11201907714UA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
SG10201913833PA (en) 2017-02-28 2020-03-30 Univ Pennsylvania Influenza vaccines based on aav vectors
AU2018228881B2 (en) 2017-03-01 2024-01-25 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
US11376321B2 (en) 2017-03-02 2022-07-05 Genethon Method for removing anti-AAV antibodies from a blood-derived composition
JP7307480B2 (ja) 2017-04-05 2023-07-12 ユニバーシティ オブ マサチューセッツ ミニ遺伝子療法
US11499154B2 (en) 2017-04-10 2022-11-15 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
IL307936A (en) 2017-04-14 2023-12-01 Regenxbio Inc Treatment for mucopolysaccharidosis 2 with the help of recombinant human iduronate-2 suplatase (ids) produced by human neuron or glial cells
WO2018200542A1 (fr) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Thérapie génique pour troubles oculaires
CN110913866A (zh) 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
WO2018208972A1 (fr) 2017-05-09 2018-11-15 University Of Massachusetts Méthodes de traitement de la sclérose latérale amyotrophique (sla)
JP7273730B2 (ja) 2017-05-11 2023-05-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 神経セロイドリポフスチン症のための遺伝子療法
CN108103058A (zh) * 2017-05-12 2018-06-01 北京五加和分子医学研究所有限公司 一种i型糖尿病的基因治疗药物
EP3621982A4 (fr) 2017-05-12 2021-05-19 University of Massachusetts Système de production de vecteurs viraux
CA3098592A1 (fr) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Therapie genique destinee au traitement de troubles des peroxysomes
BR112019025732A2 (pt) 2017-06-06 2020-06-30 University Of Massachusetts vetores de aav auto-reguladores para expressão segura de mecp2 na síndrome de rett
JP2020527335A (ja) 2017-06-14 2020-09-10 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患のための遺伝子療法
US10812532B2 (en) 2017-06-15 2020-10-20 Palo Alto Networks, Inc. Security for cellular internet of things in mobile networks
US10693918B2 (en) 2017-06-15 2020-06-23 Palo Alto Networks, Inc. Radio access technology based security in service provider networks
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US10834136B2 (en) 2017-06-15 2020-11-10 Palo Alto Networks, Inc. Access point name and application identity based security enforcement in service provider networks
US10721272B2 (en) 2017-06-15 2020-07-21 Palo Alto Networks, Inc. Mobile equipment identity and/or IOT equipment identity and application identity based security enforcement in service provider networks
US10708306B2 (en) 2017-06-15 2020-07-07 Palo Alto Networks, Inc. Mobile user identity and/or SIM-based IoT identity and application identity based security enforcement in service provider networks
US11050789B2 (en) 2017-06-15 2021-06-29 Palo Alto Networks, Inc. Location based security in service provider networks
KR20200019231A (ko) * 2017-06-22 2020-02-21 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 조절 면역 세포를 제조하기 위한 방법 및 이의 용도
SG11201912631PA (en) 2017-07-06 2020-01-30 Univ Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
WO2019008157A1 (fr) 2017-07-07 2019-01-10 Genethon Nouveaux polynucléotides codant pour une protéine fkrp humaine
WO2019018342A1 (fr) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Systeme de guide de trajectoire d'appareillage en reseau
EP3662060A2 (fr) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions et procedes de délivrance d'aav
SG11202000846WA (en) 2017-08-07 2020-02-27 Nbe Therapeutics Ag Anthracycline-based antibody drug conjugates having high in vivo tolerability
CN110709511A (zh) * 2017-08-28 2020-01-17 加利福尼亚大学董事会 腺相关病毒衣壳变体及其使用方法
RU2770922C2 (ru) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
JP7397488B2 (ja) 2017-09-22 2023-12-13 ユニバーシティ オブ マサチューセッツ Ｓｏｄ１二重発現ベクターおよびその使用
BR112020005249A2 (pt) 2017-09-22 2020-09-24 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
WO2019060726A1 (fr) 2017-09-22 2019-03-28 Genzyme Corporation Variant d'arni
JP2020537542A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症（ａｌｓ）の治療
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
AU2018350990A1 (en) 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
CA3079568A1 (fr) 2017-10-18 2019-04-25 Regenxbio Inc. Agents therapeutiques a base d'anticorps entierement humains a modification post-traductionnelle
RU2020112558A (ru) * 2017-10-18 2021-11-18 Боард Оф Регентс, Зе Юниверсити Оф Тексас Систем Терапия на основе ингибитора глутаминазы
CA3083472A1 (fr) 2017-11-27 2019-05-31 4D Molecular Therapeutics Inc. Variants de capsides de virus adeno-associe et leurs procedes d'utilisation
WO2019108856A1 (fr) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Thérapie génique pour mucopolysaccharidose de type iiib
EP3717653A4 (fr) 2017-11-30 2021-12-01 The Trustees of The University of Pennsylvania Thérapie génique pour la mucopolysaccharidose de type iiia
WO2019126329A1 (fr) 2017-12-19 2019-06-27 Akouos Llc Administration d'anticorps thérapeutiques médiée par aav à l'oreille interne
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
EP3762500A1 (fr) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Génomes aav partiels auto-complémentaires fabriqués par des cellules d'insectes
AU2019237514A1 (en) 2018-03-23 2020-10-08 University Of Massachusetts Gene therapeutics for treating bone disorders
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019204226A1 (fr) 2018-04-16 2019-10-24 University Of Massachusetts Compositions et méthodes pour l'édition génétique améliorée
CA3098448A1 (fr) 2018-04-27 2019-10-31 University Of Massachusetts Capsides de vaa identifies par la selection d'une bibliotheque in vivo
EP3788165A1 (fr) 2018-04-29 2021-03-10 REGENXBIO Inc. Systèmes et procédés de spectrophotométrie pour la détermination de contenu génomique, de contenu de capside et de rapports complets/vides de particules de virus adéno-associés
WO2019212921A1 (fr) 2018-04-29 2019-11-07 Regenxbio Inc. Procédé de clarification évolutif pour la production d'aav recombinants
US20210207168A1 (en) 2018-05-08 2021-07-08 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
EP3790627A2 (fr) 2018-05-09 2021-03-17 BioMarin Pharmaceutical Inc. Méthodes de traitement de la grippe
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
CA3099306A1 (fr) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions et methodes pour le traitement de la maladie de parkinson
EP3793616A1 (fr) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions et procédés pour l'administration de vaa
WO2019222441A1 (fr) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Sérotypes de vaa pour l'administration de charge utile spécifique au cerveau
WO2019222444A2 (fr) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Évolution dirigée
EP3806888B1 (fr) 2018-06-12 2024-01-31 Obsidian Therapeutics, Inc. Constructions régulatrices dérivées de pde5 et procédés d'utilisation en immunothérapie
EP3807404A1 (fr) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Régions 5' non traduites (5'utr) modifiées pour la production d'aav
EP3807405A2 (fr) 2018-06-14 2021-04-21 REGENXBIO Inc. Chromatographie d'échange d'anions pour la production de vaa recombinants
JP2021529513A (ja) 2018-07-02 2021-11-04 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症および脊髄に関連する障害の治療
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020026968A1 (fr) * 2018-07-30 2020-02-06 株式会社遺伝子治療研究所 Méthode pour améliorer l'expression génique par un vecteur aav
WO2020028816A1 (fr) 2018-08-03 2020-02-06 Genzyme Corporation Variante d'arni contre l'alpha-synucléine
CN112567027A (zh) 2018-08-10 2021-03-26 再生生物股份有限公司 用于重组aav生产的可扩展方法
CA3111076A1 (fr) 2018-08-30 2020-03-05 Tenaya Therapeutics, Inc. Reprogrammation de cellules cardiaques avec la myocardine et ascl1
AU2019347666A1 (en) 2018-09-28 2021-03-25 President And Fellows Of Harvard College Cellular reprogramming to reverse aging and promote organ and tissue regeneration
WO2020069461A1 (fr) 2018-09-28 2020-04-02 Voyager Therapeutics, Inc. Constructions d'expression de frataxine comprenant des promoteurs modifiés et leurs méthodes d'utilisation
WO2020069339A1 (fr) 2018-09-28 2020-04-02 President And Fellows Of Harvard College Transactivateurs de tétracycline inverses mutants pour l'expression de gènes
SG11202102942VA (en) 2018-10-01 2021-04-29 Univ Pennsylvania Compositions useful for treating gm1 gangliosidosis
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
CA3115248A1 (fr) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Constructions d'acides nucleiques modifies codant pour des proteines de production d'aav
WO2020077165A1 (fr) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions et procédés pour l'administration d'aav
CN113194982A (zh) 2018-10-12 2021-07-30 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
CN113166781A (zh) 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体
US20210324483A1 (en) 2018-10-15 2021-10-21 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
KR20210082205A (ko) 2018-10-22 2021-07-02 유니버시티 오브 로체스터 레트로바이러스 인테그라제-Cas9 융합 단백질을 이용한 유도된 비상동 DNA 삽입에 의한 게놈 편집
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
EP3650956A1 (fr) 2018-11-07 2020-05-13 Tissot S.A. Procede de diffusion d'un signal acoustique
EP3880823A4 (fr) * 2018-11-16 2022-08-17 Asklepios Biopharmaceutical, Inc. Virus adéno-associé thérapeutique pour le traitement de la maladie de pompe
KR20210093954A (ko) 2018-11-16 2021-07-28 아스텔라스세이야쿠 가부시키가이샤 유트로핀 유전자를 표적으로 하여 근이영양증을 치료하는 방법
US20220033824A1 (en) 2018-11-29 2022-02-03 University Of Massachusetts Modulation of sptlc1 via recombinant adeno-associated vectors
US20220089670A1 (en) 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
JP2022516283A (ja) 2019-01-04 2022-02-25 ウルトラジェニックス ファーマシューティカル インコーポレイテッド ウイルソン病を処置するための遺伝子治療構築物
CN113544267A (zh) 2019-01-14 2021-10-22 罗切斯特大学 使用CRISPR-Cas进行靶向核RNA裂解和聚腺苷酸化
AU2020208467A1 (en) 2019-01-18 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing AAV particles
CN113924115A (zh) 2019-01-31 2022-01-11 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
CN113710281A (zh) 2019-02-22 2021-11-26 宾夕法尼亚州大学信托人 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒
EP3927380A1 (fr) 2019-02-22 2021-12-29 University of Massachusetts Thérapie génique avec oxr1
CR20210444A (es) 2019-02-25 2021-11-02 Novartis Ag Composiciones y métodos para tratar distrofia cristalina de bietti
WO2020174369A2 (fr) 2019-02-25 2020-09-03 Novartis Ag Compositions et procédés pour traiter une dystrophie cristalline de bietti
CA3130055A1 (fr) 2019-02-26 2020-09-03 The Trustees Of The University Of Pennsylvania Compositions utiles dans le traitement de la maladie de krabbe
US20220047618A1 (en) 2019-02-28 2022-02-17 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
US20220204574A1 (en) 2019-03-25 2022-06-30 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
EP3946467A1 (fr) 2019-04-03 2022-02-09 REGENXBIO Inc. Thérapie génique pour pathologies de l'?il
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
FI3953483T3 (fi) 2019-04-11 2023-11-30 Regenxbio Inc Kokoekskluusiokromatografiamenetelmiä rekombinanttien adenoassosioituneiden viruskoostumusten karakterisoimiseksi
MX2021012540A (es) 2019-04-17 2022-01-18 Codiak Biosciences Inc Composiciones de exosomas y virus adenoasociados (aav).
TW202332458A (zh) 2019-04-19 2023-08-16 美商銳進科斯生物股份有限公司 腺相關病毒載體調配物及方法
EP3959323A1 (fr) 2019-04-24 2022-03-02 REGENXBIO Inc. Agents thérapeutiques à base d'anticorps entièrement humains à modification post-traductionnelle
TW202106879A (zh) 2019-04-29 2021-02-16 美商航海家醫療公司 於生物反應器中生產經桿狀病毒感染之昆蟲細胞(biic)之系統及方法
MX2021013266A (es) * 2019-04-29 2021-11-17 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen.
WO2020227515A1 (fr) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions et méthodes d'augmentation vectorisée de la destruction, de l'expression et/ou de la régulation de protéines
PE20212357A1 (es) * 2019-05-24 2021-12-17 Regeneron Pharma Particulas virales modificadas y usos de estas
MX2021014478A (es) 2019-05-28 2022-01-06 Astellas Pharma Inc Metodo para tratar distrofia muscular por direccionamiento del gen dmpk.
WO2020257684A1 (fr) 2019-06-20 2020-12-24 University Of Massachusetts Compositions et méthodes pour l'édition génomique améliorée
CA3145662A1 (fr) 2019-07-02 2021-01-07 M6P Therapeutics Compositions de vecteurs et leurs procedes d'utilisation pour le traitement de troubles du stockage lysosomal
US20220251567A1 (en) 2019-07-10 2022-08-11 Inserm (Institut National De La Santè Et De La Recherche Médicale) Methods for the treatment of epilepsy
US20230137971A1 (en) 2019-07-11 2023-05-04 Tenaya Therapeutics Inc. Cardiac cell reprogramming with micrornas and other factors
CA3146718A1 (fr) 2019-07-11 2021-01-14 Centre National De La Recherche Scientifique Virus adeno-associes chimiquement modifies
US10557149B1 (en) * 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
US10653731B1 (en) * 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
JP2022544015A (ja) 2019-07-23 2022-10-17 ユニバーシティ オブ ロチェスター ＣＲＩＳＰＲ－Ｃａｓでの標的化されたＲＮＡ切断
WO2021021674A1 (fr) 2019-07-26 2021-02-04 Akouos, Inc. Méthodes de traitement de la perte auditive à l'aide d'une protéine cible sécrétée
EP4004214A1 (fr) 2019-07-26 2022-06-01 RegenxBio Inc. Élément régulateur d'acide nucléique modifié et ses procédés d'utilisation
WO2021030125A1 (fr) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Milieu de culture cellulaire destiné à être utilisé dans la production de produits de thérapie génique dans des bioréacteurs
WO2021030764A1 (fr) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Variants capsidiques d'aav pour thérapie génique
WO2021041485A1 (fr) 2019-08-26 2021-03-04 Voyager Therapeutics, Inc. Expression contrôlée de protéines virales
MX2022002366A (es) 2019-08-26 2022-07-19 Regenxbio Inc Tratamiento de la retinopatía diabética con fab anti-vegf completamente humano modificado postraduccionalmente.
EP4022057A1 (fr) 2019-08-27 2022-07-06 Vertex Pharmaceuticals Incorporated Compositions et procédés pour le traitement de troubles associés à l'adn répétitif
WO2021046155A1 (fr) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Édition vectorisée d'acides nucléiques pour corriger des mutations manifestes
US20220348937A1 (en) 2019-09-06 2022-11-03 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
CN114555814A (zh) 2019-09-13 2022-05-27 罗特格斯新泽西州立大学 Aav相容的层粘连蛋白-连接子聚合蛋白
US20230270885A2 (en) 2019-09-19 2023-08-31 Genethon Gene therapy expression system alleviating cardiac toxicity of fkrp
WO2021062096A1 (fr) 2019-09-26 2021-04-01 Massachusetts Institute Of Technology Portes logiques à base de microarn et leurs utilisations
WO2021062092A1 (fr) 2019-09-26 2021-04-01 Massachusetts Institute Of Technology Arn fonctionnel trans-activé par déplacement de brin et ses utilisations
EP4038194A1 (fr) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Procédés pour une utilisation thérapeutique améliorée d'aav recombinant
JP2022552262A (ja) 2019-10-07 2022-12-15 リジェネックスバイオ インコーポレイテッド アデノ随伴ウイルスベクター医薬組成物および方法
WO2021076656A1 (fr) 2019-10-15 2021-04-22 University Of Massachusetts Trans-épissage d'arn amélioré par éditeur d'arn
US20220403414A1 (en) * 2019-10-16 2022-12-22 Wuxi Apptec (Shanghai) Co., Ltd. Novel aav variant
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療ｃ型尼曼—匹克病之腺相關病毒載體
CA3161154A1 (fr) 2019-11-14 2021-05-20 Biomarin Pharmaceutical Inc. Traitement de l'angio-oedeme hereditaire avec des vecteurs de therapie genique specifiques du foie
WO2021099394A1 (fr) 2019-11-19 2021-05-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Oligonucléotides antisens et leur utilisation pour le traitement du cancer
BR112022009895A2 (pt) 2019-11-28 2022-08-09 Regenxbio Inc Construtos de terapia de gene de microdistrofina e usos dos mesmos
KR20220112283A (ko) 2019-12-10 2022-08-10 다케다 야쿠힌 고교 가부시키가이샤 Hunter 질환 치료용 아데노-연합된 바이러스 벡터
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CA3167685A1 (fr) 2020-01-22 2021-07-29 Regenxbio Inc. Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylee entierement humaine (idua)
US20230088992A1 (en) 2020-01-29 2023-03-23 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
KR20220133900A (ko) 2020-01-29 2022-10-05 리젠엑스바이오 인크. 점액다당류증 iva의 치료
CA3165713A1 (fr) 2020-01-30 2021-08-05 Andrew Scharenberg Activateur de transduction bispecifique
TW202140781A (zh) 2020-02-02 2021-11-01 賓州大學委員會 有用於治療gm1神經節苷脂症之組成物
CA3168903A1 (fr) 2020-02-07 2021-08-12 University Of Rochester Assemblage et expression d'arn a mediation par ribozyme
US20230078498A1 (en) 2020-02-07 2023-03-16 University Of Rochester Targeted Translation of RNA with CRISPR-Cas13 to Enhance Protein Synthesis
AU2021218703A1 (en) 2020-02-14 2022-07-28 Ultragenyx Pharmaceutical Inc. Gene therapy for treating CDKL5 deficiency disorder
KR20220149590A (ko) 2020-03-02 2022-11-08 테나야 테라퓨틱스, 인코포레이티드 심근세포-발현 마이크로rna에 의한 유전자 벡터 제어
EP4127169A1 (fr) 2020-03-27 2023-02-08 University of Rochester Destruction ciblée d'arn viral par crispr-cas13
US20230103771A1 (en) 2020-03-27 2023-04-06 University Of Rochester CRISPR-Cas13 crRNA Arrays
AU2021245031A1 (en) 2020-03-27 2022-11-03 UCB Biopharma SRL Autonomous knob domain peptides
WO2021202532A1 (fr) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Thérapie génique pour le traitement de l'acidémie propionique
CA3177182A1 (fr) 2020-03-31 2021-10-07 University Of Massachusetts Variants capsidiques de vaa et leurs utilisations
AR122409A1 (es) 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
BR112022020444A2 (pt) 2020-04-10 2022-11-29 Sola Biosciences Llc Composições e métodos para o tratamento de distúrbios de agregação de proteínas
EP4135841A1 (fr) 2020-04-15 2023-02-22 Voyager Therapeutics, Inc. Composés de liaison à la protéine tau
JP2023524414A (ja) 2020-04-28 2023-06-12 ソラ・バイオサイエンシズ・エルエルシー Ｔｄｐ－４３プロテイノパチーの処置のための組成物および方法
TW202208622A (zh) 2020-05-12 2022-03-01 賓州大學委員會 用於治療克拉培氏病之組成物
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CA3183171A1 (fr) 2020-05-13 2021-11-18 Akouos, Inc. Compositions et methodes pour traiter une perte auditive associee a slc26a4
AR122079A1 (es) 2020-05-13 2022-08-10 Akouos Inc Composiciones y métodos para tratar la perdida auditiva asociada a kcnq4
WO2021230385A1 (fr) 2020-05-15 2021-11-18 Astellas Pharma Inc. Procédé de traitement de la dystrophie musculaire par ciblage d'un gène utrophine
WO2021247995A2 (fr) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la douleur neuropathique
CN116096737A (zh) 2020-06-05 2023-05-09 索拉生物科学有限公司 用于治疗突触核蛋白病的组合物和方法
AU2021292200A1 (en) 2020-06-17 2023-02-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
WO2021255245A2 (fr) 2020-06-19 2021-12-23 Genethon Système d'expression de thérapie génique permettant une expression adéquate dans les muscles et dans le cœur de sgcg
CN116194587A (zh) 2020-07-10 2023-05-30 吉尼松公司 一种新的肌肉特异性启动子
WO2022011262A1 (fr) 2020-07-10 2022-01-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Méthodes et compositions pour le traitement de l'épilepsie
KR20230038503A (ko) 2020-07-13 2023-03-20 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 샤르코-마리-투스 질환의 치료에 유용한 조성물
WO2022015916A1 (fr) 2020-07-15 2022-01-20 University Of Rochester Clivage d'arn ciblé avec des protéines de fusion dcasl3-rnase
WO2022017630A1 (fr) 2020-07-23 2022-01-27 Ucl Business Ltd Vecteur de thérapie génique pour eef1a2 et ses utilisations
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4192514A1 (fr) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Milieu de culture cellulaire destiné à être utilisé dans la production de produits de thérapie génique dans des bioréacteurs
US20240043494A1 (en) 2020-08-07 2024-02-08 Amicus Therapeutics, Inc. Vesicle Targeting Proteins And Uses Of Same
CN116194154A (zh) 2020-08-07 2023-05-30 太空飞船七有限责任公司 使用aav载体的plakophilin-2(pkp2)基因疗法
CN114075610B (zh) * 2020-08-11 2023-11-17 北京荷塘生华医疗科技有限公司 检测野生型腺相关病毒的通用引物及其应用
CN116209769A (zh) 2020-08-14 2023-06-02 宾夕法尼亚州大学信托人 新型aav衣壳和含有新型aav衣壳的组合物
EP4200429A1 (fr) 2020-08-24 2023-06-28 The Trustees of The University of Pennsylvania Vecteurs viraux codant pour des fusions d'agonistes du récepteur glp-1 et leurs utilisations dans le traitement de maladies métaboliques
GB202013194D0 (en) 2020-08-24 2020-10-07 Combigene Ab Gene therapy for lipodystrophy
KR20230058102A (ko) 2020-08-26 2023-05-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn 관련 성인 발병 신경변성 치료를 위한 재조합 아데노-연관 바이러스
WO2022056000A1 (fr) 2020-09-09 2022-03-17 Vertex Pharmaceuticals Incorporated Compositions et méthodes de traitement de la dystrophie musculaire de duchenne
JP2023540960A (ja) 2020-09-10 2023-09-27 ルートビヒ－マキシミリアンズ－ウニベルジテート ミュンヘン 操作されたａａｖベクター
EP4213890A1 (fr) 2020-09-15 2023-07-26 RegenxBio Inc. Lanadelumab vectorisé et administration de celui-ci
WO2022060916A1 (fr) 2020-09-15 2022-03-24 Regenxbio Inc. Anticorps vectorisés pour thérapie antivirale
WO2022061002A1 (fr) 2020-09-18 2022-03-24 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Nouveaux vecteurs viraux adéno-associés (vaa) pour traiter l'acidémie méthylmalonique (amm) héréditaire provoquée par une déficience en méthylmalonyl-coa mutase (mmut)
EP4217376A1 (fr) 2020-09-24 2023-08-02 University of Massachusetts Vecteurs vaa codant pour nf1 et leurs utilisations
TW202229559A (zh) 2020-10-01 2022-08-01 美商健臻公司 藉由肝導向基因替代療法治療pku之人類pah表現匣
WO2022076549A1 (fr) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations pour administration suprachoroïdienne, telles que formulations à viscosité élevée
WO2022076750A2 (fr) 2020-10-07 2022-04-14 Regenxbio Inc. Virus adéno-associés recombinants pour administration musculaire ou dans le snc
CA3198368A1 (fr) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations pour administration suprachoroidienne, telles que formulations avec formation d'agregats
CA3197342A1 (fr) 2020-10-07 2022-04-14 Regenxbio Inc. Therapie genique pour manifestations oculaires de maladie cln2
AU2021356645A1 (en) 2020-10-07 2023-05-25 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
WO2022076711A2 (fr) 2020-10-07 2022-04-14 Regenxbio Inc. Virus adéno-associés pour administration oculaire de thérapie génique
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
KR20230118075A (ko) 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
AR123838A1 (es) 2020-10-18 2023-01-18 Univ Pennsylvania Vector mejorado de virus adenoasociado (aav) y usos de este
WO2022094157A1 (fr) 2020-10-28 2022-05-05 Regenxbio Inc. Anticorps vectorisés anti-cgrp et anti-récepteur de cgrp et leur administration
CA3195967A1 (fr) 2020-10-28 2022-05-05 Xu Wang Anticorps anti-tnf-? vectorises pour indications oculaires
WO2022094078A1 (fr) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions utiles dans le traitement du syndrome de rett
CA3196964A1 (fr) 2020-10-29 2022-05-05 Xu Wang Antagonistes de tnf-alpha vectorises pour des indications oculaires
EP4236974A2 (fr) 2020-10-29 2023-09-06 RegenxBio Inc. Anticorps de facteur xii vectorisés et leur administration
US20230414648A1 (en) 2020-11-06 2023-12-28 Vertex Pharmaceuticals Incorporated Compositions and Methods for Treatment of DM1 with SLUCAS9 and SACAS9
CN112322791B (zh) * 2020-11-27 2023-10-27 福建省农业科学院畜牧兽医研究所 一种新型鸭依赖属病毒环介导等温扩增检测引物组及试剂盒
TW202237644A (zh) 2020-12-01 2022-10-01 美商阿科奧斯公司 用於治療前庭神經鞘瘤相關症狀之抗ｖｅｇｆ抗體構築體及相關方法
US20240024507A1 (en) 2020-12-01 2024-01-25 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
JP2023551911A (ja) 2020-12-01 2023-12-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アンジェルマン症候群の治療のための組成物及びその使用
AU2021403076A1 (en) 2020-12-16 2023-06-29 Regenxbio Inc. Method of producing a recombinant adeno-associated virus particle
TW202241943A (zh) 2020-12-29 2022-11-01 美商銳進科斯生物股份有限公司 Tau特異性抗體基因療法組合物、方法及其用途
IL304006A (en) 2020-12-29 2023-08-01 Akouos Inc Compositions and methods for treating hearing loss and/or vision loss associated with CLRN1
CA3205209A1 (fr) 2021-01-21 2022-07-28 Regenxbio Inc. Production amelioree de polypeptides et de virus recombines
BR112023014980A2 (pt) 2021-01-27 2023-10-10 Umoja Biopharma Inc Lentivírus para gerar células que expressam o receptor de antígeno quimérico anti-cd19
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
KR20230145386A (ko) 2021-02-10 2023-10-17 리젠엑스바이오 인크. 재조합 인간 이두로네이트-2-설파타아제(ids)를 사용한 점액다당류증 ii의 치료
GB202101958D0 (en) 2021-02-12 2021-03-31 Ucl Business Ltd Gene therapy for dopamine transporter deficiency syndrome
WO2022182957A1 (fr) 2021-02-26 2022-09-01 Vertex Pharmaceuticals Incorporated Compositions et méthodes de traitement de la dystrophie myotonique de type 1 avec crispr/sacas9
EP4298227A1 (fr) 2021-02-26 2024-01-03 Takeda Pharmaceutical Company Limited Composition et méthodes de traitement de la maladie de fabry
EP4298221A1 (fr) 2021-02-26 2024-01-03 Vertex Pharmaceuticals Incorporated Compositions et méthodes pour le traitement de la dystrophie myotonique de type 1 avec crispr/slucas9
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN117460832A (zh) 2021-03-22 2024-01-26 建新公司 空aav衣壳和完整aav衣壳的尺寸排阻色谱分析
WO2022204476A1 (fr) 2021-03-26 2022-09-29 The Board Of Regents Of The University Of Texas System Édition de nucléotides pour remettre en phase des transcrits de la dmd par édition de base et édition génomique prémium (« prime editing »)
AU2022258312A1 (en) 2021-04-12 2023-10-26 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
EP4326753A2 (fr) 2021-04-23 2024-02-28 University of Rochester Édition génomique par insertion d'adn non homologue dirigée à l'aide d'une protéine de fusion cas-intégrase rétrovirale et méthodes de traitement
JP2024517143A (ja) 2021-04-26 2024-04-19 リジェネックスバイオ インコーポレイテッド ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与
WO2022229851A1 (fr) 2021-04-26 2022-11-03 Crispr Therapeutics Ag Compositions et procédés d'utilisation de séquences d'échafaudage slucas9
EP4334454A2 (fr) 2021-05-04 2024-03-13 RegenxBio Inc. Nouveaux vecteurs aav et procédés et utilisations associés
WO2022234519A1 (fr) 2021-05-05 2022-11-10 Crispr Therapeutics Ag Compositions et méthodes d'utilisation de séquences d'échafaudage sacas9
WO2022241030A1 (fr) 2021-05-11 2022-11-17 Regenxbio Inc. Traitement de la dystrophie musculaire de duchenne et combinaisons correspondantes
AU2022299552A1 (en) 2021-06-25 2024-01-04 Oxford Biomedica (Us) Llc Adeno-associated virus packaging systems
CA3225985A1 (fr) 2021-07-01 2023-01-05 Indapta Therapeutics, Inc. Cellules tueuses naturelles (nk) modifiees et methodes associees
EP4363437A1 (fr) 2021-07-01 2024-05-08 Amicus Therapeutics, Inc. Variants de neurotensine et protéines marquées comprenant un propeptide de neurotensine ou de sortiline
IL309742A (en) 2021-07-08 2024-02-01 Tenaya Therapeutics Inc Optimal expression cassettes for gene therapy
EP4373947A1 (fr) 2021-07-19 2024-05-29 New York University Polythérapies auf1 pour le traitement d'une maladie dégénérative musculaire
EP4377459A2 (fr) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions et procédés de modulation de l'expression de la frataxine
WO2023010135A1 (fr) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions et procédés pour moduler l'expression de la protéine 2 de liaison méthyle-cpg (mecp2)
WO2023018637A1 (fr) 2021-08-09 2023-02-16 Vertex Pharmaceuticals Incorporated Édition génique d'éléments régulateurs
CA3227108A1 (fr) 2021-08-11 2023-02-16 Xiaomeng HU Cellules primaires genetiquement modifiees pour une therapie cellulaire allogenique
AU2022326565A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
WO2023019227A1 (fr) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Cellules génétiquement modifiées pour une thérapie cellulaire allogénique pour réduire les réactions inflammatoires induites par le complément
AU2022325955A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
WO2023039444A2 (fr) 2021-09-08 2023-03-16 Vertex Pharmaceuticals Incorporated Excision précise de parties de l'exon 51 pour le traitement de la dystrophie musculaire de duchenne
IL311572A (en) 2021-09-30 2024-05-01 Akouos Inc Compositions and methods for treating KCNQ4-related hearing loss
AR127217A1 (es) 2021-10-01 2023-12-27 Biomarin Pharm Inc Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas
WO2023056399A1 (fr) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Nouvelles capsides de vaa et compositions les contenant
WO2023060113A1 (fr) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions et procédés de production d'aav recombinants
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060269A1 (fr) 2021-10-07 2023-04-13 Regenxbio Inc. Virus adéno-associés recombinants pour administration ciblée
WO2023060272A2 (fr) 2021-10-07 2023-04-13 Regenxbio Inc. Virus adéno-associés recombinants pour administration tropique au snc
WO2023060221A2 (fr) 2021-10-08 2023-04-13 Sola Biosciences Llc Compositions et méthodes de traitement de protéopathies
CA3234720A1 (fr) 2021-10-08 2023-04-13 Sola Biosciences Llc Compositions et methodes de traitement de cancers a mediation par p53
AU2022371442A1 (en) 2021-10-21 2024-04-18 Vertex Pharmaceuticals Incorporated Hypoimmune cells
WO2023077092A1 (fr) 2021-10-28 2023-05-04 Regenxbio Inc. Éléments régulateurs d'acides nucléiques modifiés, procédés et utilisations de ceux-ci
WO2023087019A2 (fr) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions pour la réduction spécifique de drg de l'expression de transgènes
WO2023102442A1 (fr) 2021-11-30 2023-06-08 Jaguar Gene Therapy, Llc Procédés de thérapie génique pour le traitement du diabète
WO2023102517A1 (fr) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions et méthodes de traitement de la maladie de fabry
WO2023111348A1 (fr) 2021-12-17 2023-06-22 Centre National De La Recherche Scientifique Peptides et procédés destinés à être utilisés dans le traitement de la douleur
WO2023133593A2 (fr) * 2022-01-10 2023-07-13 University Of Florida Research Foundation, Incorporated Variants capsidiques de vaa5
WO2023133574A1 (fr) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions et méthodes utiles pour le traitement de troubles médiés par c9orf72
WO2023133584A1 (fr) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions utiles dans le traitement d'une leucodystrophie métachromatique
WO2023133595A2 (fr) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Méthodes de dosage et d'administration ex vivo de particules lipidiques ou de vecteurs viraux ainsi que systèmes et utilisations associés
EP4215614A1 (fr) 2022-01-24 2023-07-26 Dynacure Thérapie de combinaison pour maladies liées à la dystrophine
WO2023147304A1 (fr) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Capsides d'aav pour une transduction cardiaque améliorée et un ciblage du foie
WO2023150647A1 (fr) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Procédés d'administration et de dosage répétés de particules lipidiques ou de vecteurs viraux et systèmes et utilisations connexes
TW202342525A (zh) 2022-02-02 2023-11-01 美商阿科奧斯公司 用於治療前庭神經鞘瘤相關症狀之抗ｖｅｇｆ抗體構築體及相關方法
TW202346599A (zh) 2022-02-08 2023-12-01 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
WO2023158836A1 (fr) 2022-02-17 2023-08-24 Sana Biotechnology, Inc. Protéines cd47 modifiées et leurs utilisations
WO2023156530A1 (fr) 2022-02-17 2023-08-24 Lysogene Thérapie génique pour maladies neurodégénératives
TW202345911A (zh) 2022-03-08 2023-12-01 美商維泰克斯製藥公司 用於治療杜興氏肌肉失養症(duchenne muscular dystrophy)之部分外顯子44、50及53之精確切除
WO2023172926A1 (fr) 2022-03-08 2023-09-14 Vertex Pharmaceuticals Incorporated Excisions précises de parties d'exons pour le traitement de la dystrophie musculaire de duchenne
WO2023173123A1 (fr) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Cellules génétiquement modifiées et compositions et utilisations associées
WO2023178053A1 (fr) 2022-03-13 2023-09-21 Regenxbio Inc. Promoteurs spécifiques à un muscle modifiés
WO2023183623A1 (fr) 2022-03-25 2023-09-28 Regenxbio Inc. Thérapie génique du virus adéno-associé à facteur de nécrose tumorale alpha dominant-négatif
WO2023187728A1 (fr) 2022-04-01 2023-10-05 Takeda Pharmaceutical Company Limited Thérapie génique pour des maladies avec des manifestations du snc
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
WO2023196892A1 (fr) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Immunisation passive avec des anticorps neutralisants anti-aav pour empêcher la transduction hors cible de vecteurs aav administrés par voie intrathécale
US20230365968A1 (en) 2022-04-06 2023-11-16 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2023196851A1 (fr) 2022-04-06 2023-10-12 President And Fellows Of Harvard College Inversion du vieillissement du système nerveux central
WO2023196873A1 (fr) 2022-04-06 2023-10-12 Regenxbio Inc. Composition pharmaceutique comprenant un vecteur de virus adéno-associé recombinant avec une cassette d'expression codant un transgène pour administration suprachoroïdienne
WO2023196893A1 (fr) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions et méthodes de traitement d'un cancer du sein métastatique her2 positif et d'autres cancers
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
WO2023201207A1 (fr) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Virus adéno-associé comprenant une capside modifiée
WO2023198652A1 (fr) 2022-04-11 2023-10-19 Centre National De La Recherche Scientifique Virus adéno-associés chimiquement modifiés
US20230346977A1 (en) 2022-04-13 2023-11-02 Universitat Autònoma De Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
WO2023201277A1 (fr) 2022-04-14 2023-10-19 Regenxbio Inc. Virus adéno-associés recombinants pour administration tropique au snc
WO2023201308A1 (fr) 2022-04-14 2023-10-19 Regenxbio Inc. Thérapie génique pour le traitement d'une maladie oculaire
WO2023205606A1 (fr) 2022-04-18 2023-10-26 Vertex Pharmaceuticals Incorporated Compositions et procédés pour améliorer la thérapie par vaa et diminuer le tropisme de vaa vers le foie
WO2023215807A1 (fr) 2022-05-03 2023-11-09 Regenxbio Inc. INHIBITEURS VECTORISÉS ANTI-TNF-α POUR INDICATIONS OCULAIRES
TW202400803A (zh) 2022-05-03 2024-01-01 美商銳進科斯生物股份有限公司 載體化抗補體抗體與補體劑及其投與
WO2023214346A1 (fr) 2022-05-06 2023-11-09 Novartis Ag Nouveaux polypeptides de fusion vp2 d'aav recombinants
WO2023227731A1 (fr) 2022-05-25 2023-11-30 Tafalgie Therapeutics Peptides et procédés destinés à être utilisés dans le traitement de la douleur
WO2023239627A2 (fr) 2022-06-08 2023-12-14 Regenxbio Inc. Méthodes de production d'aav recombinant
WO2023240236A1 (fr) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement de troubles liés à l'amyotrophie spinale
TW202409289A (zh) * 2022-06-27 2024-03-01 美商安斯泰來基因治療股份有限公司 用於治療肌強直性營養不良之組合物及方法
WO2024006741A1 (fr) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. Variants de capside d'aav et leurs utilisations
WO2024007020A1 (fr) 2022-06-30 2024-01-04 Indapta Therapeutics, Inc. Combinaison de cellules tueuses naturelles (nk) modifiées et d'une thérapie par anticorps et méthodes associées
WO2024011112A1 (fr) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Variants de capside d'aav et leurs utilisations
WO2024015881A2 (fr) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. Compositions, systèmes et procédés d'activation transcriptionnelle ciblée
WO2024020352A1 (fr) 2022-07-18 2024-01-25 Vertex Pharmaceuticals Incorporated Arn guides tandem (arntg) et leur utilisation dans l'édition génomique
WO2024017990A1 (fr) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Méthodes et compositions pour le traitement de troubles de la douleur chronique
WO2024026377A1 (fr) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Procédés de transduction utilisant un vecteur viral et des inhibiteurs de facteurs de restriction antiviraux
CN115354049A (zh) * 2022-07-29 2022-11-18 中国科学院深圳先进技术研究院 一种基因递送系统在将目的基因经静脉注射递送至肝脏的应用
WO2024044725A2 (fr) 2022-08-24 2024-02-29 Regenxbio Inc. Virus adéno-associés recombinants et leurs utilisations
WO2024042485A1 (fr) 2022-08-25 2024-02-29 Takeda Pharmaceutical Company Limited Composition destinée à être utilisée dans le traitement de la maladie de fabry
WO2024054983A1 (fr) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Expression controlée de protéines virales
WO2024064863A2 (fr) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Traitement de la cardiomyopathie arythmogène avec des vecteurs de thérapie génique aav
WO2024064856A1 (fr) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Traitement de la cardiomyopathie au moyen de vecteurs de thérapie génique aav
WO2024073669A1 (fr) 2022-09-30 2024-04-04 Regenxbio Inc. Traitement de maladies oculaires avec des vecteurs viraux recombinés codant un fab anti-vegf
WO2024081746A2 (fr) 2022-10-11 2024-04-18 Regenxbio Inc. Éléments régulateurs d'acides nucléiques modifiés, méthodes et utilisations de ceux-ci
WO2024105638A1 (fr) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Vecteurs aav recombinants et méthodes de traitement de la maladie de hunter
WO2024130067A2 (fr) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Vecteurs mutants aav recombinants avec motifs de ciblage spécifiques des muscles cardiaque et squelettique et compositions les contenant
WO2024130070A2 (fr) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Capsides aav recombinantes avec motifs de ciblage spécifiques du muscle cardiaque et squelettique et leurs utilisations
CN116622908B (zh) * 2023-04-13 2024-02-06 武汉珈创生物技术股份有限公司 快速检测野生型腺相关病毒的引物探针、试剂盒及方法和应用

Family Cites Families (70)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8929110D0 (en) * 1989-12-22 1990-02-28 3I Res Expl Ltd Polypeptides and dna encoding therefor
US5449616A (en) 1990-05-23 1995-09-12 University Of Iowa Research Foundation Nucleic acid encoding dystrophin-associated protein
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
CA2046745A1 (fr) 1991-07-10 1993-01-11 Isaac Neuman Article forme d'un boitier pour au moins une pierre precieuse
US6174666B1 (en) 1992-03-27 2001-01-16 The United States Of America As Represented By The Department Of Health And Human Services Method of eliminating inhibitory/instability regions from mRNA
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US6001650A (en) * 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
JPH11514853A (ja) * 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
ATE264398T1 (de) * 1995-12-01 2004-04-15 Crucell Holland Bv Regulierte expression von proteinen in stabil transformierten säugetierzellen
EP0950111A1 (fr) * 1996-09-06 1999-10-20 The Trustees Of The University Of Pennsylvania Procede d'utilisation de cre-lox pour la production de virus adeno-associes de recombinaison
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
JP2001500015A (ja) 1996-09-06 2001-01-09 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
WO1998010087A1 (fr) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Vecteurs d'adenovirus de chimpanze
ATE380037T1 (de) 1996-09-06 2007-12-15 Univ Pennsylvania Verfaheren zur durch rekombinante adeno- assoziierte virus-gerichtete gentherapie
AU4645697A (en) * 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
AU5070298A (en) * 1996-12-05 1998-06-29 Introgene B.V. Genetic modification of primate hemopoietic repopulating stem cells
US6039942A (en) * 1996-12-20 2000-03-21 Novo Nordick A/S Phytase polypeptides
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
EP1015619A1 (fr) * 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Procedes et lignee cellulaire utiles pour la production de virus adeno-associes recombines
CA2303768C (fr) * 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Procedes et produits genetiques vectoriels utiles pour obtenir un virus adeno-associe (aav)
CA2304131A1 (fr) 1997-09-19 1999-04-01 James M. Wilson Procede de transfert de gene utilisant bcl2 et compositions utiles a cet effet
CA2324225A1 (fr) 1998-03-20 1999-09-23 The Trustees Of The University Of Pennsylvania Compositions et methodes de production de virus adeno-associes recombines sans auxiliaire
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
CA2745131C (fr) 1998-05-28 2016-08-09 John A. Chiorini Vecteurs d'aav5 et leurs utilisation
US6210663B1 (en) 1998-08-20 2001-04-03 The Wistar Institute Of Anatomy And Biology Methods of augmenting mucosal immunity through systemic priming and mucosal boosting
US6759237B1 (en) * 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
AU768729B2 (en) * 1998-11-05 2004-01-08 Trustees Of The University Of Pennsylvania, The Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6387368B1 (en) * 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
JP4693244B2 (ja) * 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
CA2375098A1 (fr) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions et methodes pour la fabrication de virus recombines necessitant des virus auxiliaires
EP1212411A2 (fr) * 1999-08-20 2002-06-12 The Johns Hopkins University School Of Medicine Techniques et compositions permettant de construire et d'utiliser des bibliotheques de fusion
AU1071701A (en) * 1999-09-29 2001-04-30 Trustees Of The University Of Pennsylvania, The Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
US6365394B1 (en) * 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
EP1234048A2 (fr) 1999-12-03 2002-08-28 The Trustees Of The University Of Pennsylvania Compositions et procedes servant a augmenter l'encapsidation et la production d'adenovirus de recombinaison au moyen de signaux multiples d'encapsidation
US6821512B1 (en) 1999-12-03 2004-11-23 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals
AU4565401A (en) * 2000-03-14 2001-09-24 Thomas Jefferson University Production of chimeric capsid vectors
US6468524B1 (en) * 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
AU2001255575B2 (en) * 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2001289177A1 (en) * 2000-08-30 2002-03-13 Haplogen, Llc Method for determining alleles
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US20020159978A1 (en) * 2001-02-06 2002-10-31 James Allen Muscle-directed gene transfer by use of recombinant AAV-1 and AAV-6 virions
ATE520707T1 (de) * 2001-11-13 2011-09-15 Univ Pennsylvania Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
EP2359869B1 (fr) * 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Séquences de virus adéno-associés de sérotype 8 , vecteurs les contenant et leurs utilisations
AU2002367943A1 (en) 2001-12-18 2003-12-22 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
AU2003253595A1 (en) * 2002-04-05 2003-11-03 The Children's Hospital Of Philadelphia Methods for the production of chimeric adeno-associated virus (aav) vectors, compositions of chimeric aav vectors, and methods of use thereof
CA2426283C (fr) * 2002-04-29 2006-06-27 The Trustees Of The University Of Pennsylvania Methode d'isolement et d'amplification directs de virus integres dans l'adn cellulaire de tissus
ES2862206T3 (es) 2003-06-19 2021-10-07 Genzyme Corp Viriones de AAV con inmunorreactividad disminuida y sus usos
EP2298926A1 (fr) * 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Clades (sous-types) et séquences de Virus Adeno-Associé (AAV), vecteurs les contenant et leurs utilistation
US20090054823A1 (en) 2004-09-30 2009-02-26 The Trustees Of The University Of Pennsylvania Perfusion circuit and use therein in targeted delivery of macromolecules
EP3409296A1 (fr) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Procédé d'amélioration de la fonction d'un vecteur aav
US7788045B2 (en) 2005-09-01 2010-08-31 Meditasks, Llc Systems and method for homeostatic blood states
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
JP2009102988A (ja) 2007-10-19 2009-05-14 Toyota Motor Corp 内燃機関の燃料噴射装置
CA2718495C (fr) 2008-03-14 2022-06-14 Humanzyme Limited Production recombinante de proteines humaines authentiques utilisant des systemes d'expression de cellules humaines
US20090280103A1 (en) 2008-04-04 2009-11-12 Martin Flueck Regulation of muscle repair
US8729041B2 (en) 2008-12-03 2014-05-20 The Johns Hopkins University Compositions and methods for treating hepatic neoplasia
CA3066596A1 (fr) 2010-04-23 2011-10-27 University Of Massachusetts Vecteurs aav ciblant le systeme nerveux central et leurs procedes d'utilisation
CN103648328B (zh) 2011-07-15 2016-01-06 阿尔弗雷德·凯驰两合公司 旋转刷和带旋转刷的清扫机
WO2013078316A1 (fr) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Administration de virus adéno-associé recombinant de polynucléotides alpha-sarcoglycanes
US20150111955A1 (en) 2012-02-17 2015-04-23 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
WO2013170078A1 (fr) 2012-05-09 2013-11-14 Oregon Health & Science University Plasmides et vecteurs viraux associés à un adénovirus
US9819463B2 (en) 2016-02-18 2017-11-14 Huawei Technologies Co., Ltd. Method and apparatus for transmitting data in a wireless communication system
SG11201907714UA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor

Also Published As

Publication number Publication date
HUP0600229A2 (en) 2006-06-28
NO20042183L (no) 2004-07-14
US20210285012A1 (en) 2021-09-16
CA2406745A1 (fr) 2003-05-13
CN103555678B (zh) 2018-02-09
IL221602A (en) 2015-09-24
JP4677187B2 (ja) 2011-04-27
NZ618298A (en) 2015-04-24
DE60209193T2 (de) 2006-09-28
PL404537A1 (pl) 2014-02-17
EP1456419A4 (fr) 2005-11-09
NO336468B1 (no) 2015-08-31
US8906675B2 (en) 2014-12-09
CN105671005B (zh) 2020-09-15
US20110263027A1 (en) 2011-10-27
US20200131534A1 (en) 2020-04-30
CA2945734C (fr) 2020-02-25
ATE317916T1 (de) 2006-03-15
BR122016004944B8 (pt) 2021-07-27
ES2439515T3 (es) 2014-01-23
CA2406745C (fr) 2006-01-10
BRPI0214119B1 (pt) 2016-09-27
PL373864A1 (en) 2005-09-19
CA3159060C (fr) 2023-05-16
IL234063A0 (en) 2014-09-30
US9790472B2 (en) 2017-10-17
US20180030479A1 (en) 2018-02-01
BR122016004546B1 (pt) 2016-11-29
AU2002361573B2 (en) 2008-06-12
IL227866A0 (en) 2013-09-30
US10526617B2 (en) 2020-01-07
JP2013013414A (ja) 2013-01-24
EP2341068B1 (fr) 2013-09-25
CA2945734A1 (fr) 2003-05-22
KR101014207B1 (ko) 2011-02-14
IL193525A0 (en) 2009-02-11
US20030138772A1 (en) 2003-07-24
DK1310571T3 (da) 2006-06-19
IL248724A0 (en) 2017-01-31
ES2258601T3 (es) 2006-09-01
BR122016004546B8 (pt) 2021-07-27
JP5140627B2 (ja) 2013-02-06
US11034977B2 (en) 2021-06-15
SG168422A1 (en) 2011-02-28
NO20131359L (no) 2004-07-14
NZ578982A (en) 2011-03-31
IL193525A (en) 2014-08-31
EP1456419A2 (fr) 2004-09-15
NO336801B1 (no) 2015-11-02
NZ591012A (en) 2012-06-29
EP1310571A2 (fr) 2003-05-14
CN102181480B (zh) 2016-01-27
US10041090B2 (en) 2018-08-07
CN105671005A (zh) 2016-06-15
PL409838A1 (pl) 2015-07-20
CA3159060A1 (fr) 2003-05-22
CA2864537A1 (fr) 2003-05-22
US20110151434A1 (en) 2011-06-23
JP5669795B2 (ja) 2015-02-18
HU230406B1 (hu) 2016-04-28
US20160097040A1 (en) 2016-04-07
CA3066428C (fr) 2022-05-24
NO20150196L (no) 2004-07-14
EP1310571A3 (fr) 2003-08-13
US20200087684A1 (en) 2020-03-19
EP2338900A1 (fr) 2011-06-29
ZA200403360B (en) 2005-04-26
US20170240921A1 (en) 2017-08-24
JP2005525086A (ja) 2005-08-25
CN103555676B (zh) 2016-08-31
CA2915124C (fr) 2018-08-14
NZ564506A (en) 2009-09-25
MX346493B (es) 2017-03-21
CN103555677B (zh) 2018-01-30
JP2016136953A (ja) 2016-08-04
CN103555677A (zh) 2014-02-05
PH12016502583A1 (en) 2017-10-18
HK1068925A1 (en) 2005-05-06
IL213810A (en) 2014-08-31
IL258545B (en) 2019-11-28
IL233391A0 (en) 2014-08-31
IL270065A (fr) 2019-12-31
CA2756866C (fr) 2017-06-06
IL227866A (en) 2015-11-30
CN103555676A (zh) 2014-02-05
EP1456419B1 (fr) 2011-08-17
IL161827A (en) 2009-11-18
DE60209193D1 (de) 2006-04-20
PL220644B1 (pl) 2015-11-30
US10508286B2 (en) 2019-12-17
CA2915124A1 (fr) 2003-05-22
PL217623B1 (pl) 2014-08-29
NO20140988L (no) 2004-07-14
US8524446B2 (en) 2013-09-03
IL233391A (en) 2016-12-29
US11499167B2 (en) 2022-11-15
IL270065B1 (en) 2023-06-01
US10308958B2 (en) 2019-06-04
HUP0600229A3 (en) 2010-01-28
CN103589692B (zh) 2018-07-24
SG10201912509RA (en) 2020-02-27
CN103589692A (zh) 2014-02-19
JP2014239680A (ja) 2014-12-25
WO2003042397A2 (fr) 2003-05-22
US10544432B2 (en) 2020-01-28
ATE520707T1 (de) 2011-09-15
PL221877B1 (pl) 2016-06-30
CA2756866A1 (fr) 2003-05-22
PH12014501487B1 (en) 2015-12-07
HK1056198A1 (en) 2004-02-06
IL270065B2 (en) 2023-10-01
CN102181480A (zh) 2011-09-14
ES2455126T3 (es) 2014-04-14
IL234063A (en) 2017-06-29
US20220025401A1 (en) 2022-01-27
JP2003235562A (ja) 2003-08-26
US20190024117A1 (en) 2019-01-24
US20200080109A1 (en) 2020-03-12
BR0214119A (pt) 2005-06-28
US11041171B2 (en) 2021-06-22
KR20100029134A (ko) 2010-03-15
IL258545A (en) 2018-06-28
US20220213501A1 (en) 2022-07-07
EP2341068A1 (fr) 2011-07-06
BR122016004944B1 (pt) 2016-11-22
CA2465868C (fr) 2017-02-28
IL221602A0 (en) 2012-10-31
MX359371B (es) 2018-09-25
PL397823A1 (pl) 2012-07-16
JP2009195237A (ja) 2009-09-03
KR20050058234A (ko) 2005-06-16
NZ532635A (en) 2007-05-31
KR101015854B1 (ko) 2011-02-23
IL213810A0 (en) 2011-07-31
US20170298388A1 (en) 2017-10-19
NO338362B1 (no) 2016-08-15
HU229379B1 (en) 2013-11-28
US11377669B2 (en) 2022-07-05
EP1310571B1 (fr) 2006-02-15
SG10201506627PA (en) 2015-10-29
SG10202108118RA (en) 2021-08-30
SG157224A1 (en) 2009-12-29
CN103555678A (zh) 2014-02-05
NZ600121A (en) 2013-12-20
WO2003042397A3 (fr) 2004-02-05
MXPA04004600A (es) 2004-08-13
PL222683B1 (pl) 2016-08-31
BRPI0214119B8 (pt) 2021-05-25
JP3958191B2 (ja) 2007-08-15
CA2864537C (fr) 2016-11-29
US11034976B2 (en) 2021-06-15
NZ548094A (en) 2008-07-31
JP6212142B2 (ja) 2017-10-11
JP5969547B2 (ja) 2016-08-17
CA2465868A1 (fr) 2003-05-22
US20130045186A1 (en) 2013-02-21
CA3066428A1 (fr) 2003-05-22
EP2338900B1 (fr) 2014-01-01
IL248724B (en) 2018-04-30
NO334379B1 (no) 2014-02-24
PH12014501487A1 (en) 2015-12-07

Similar Documents

Publication Publication Date Title
IL248724A0 (en) A method for tracking and/or identifying sequences of adenoid virus (aav) and isolating new sequences so identified
WO2003064993A3 (fr) Methodes et materiels de detection de materiel genetique
WO2002059379A3 (fr) Methodes de detection, de graduation ou de surveillance d'une infection a $i(h. pylori)
DK0974658T3 (da) Prober til diagnose af infektioner forårsaget af Streptococcus pyogenes
WO2001057209A3 (fr) Nouvelle famille de proteines, denommees atip, les sequences nucleiques codant pour lesdites proteines et leurs applications
DK0972833T3 (da) Prober til diagnose af infektioner forårsaget af Klebsiella pneumoniae
WO2003052048A3 (fr) Proteines liees a des acides nucleiques
WO2002074913A3 (fr) Proteines associees a des acides nucleiques
WO2003050294A3 (fr) Analyse genotypique au moyen de tige capillaire humaine