ATE520707T1 - Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen - Google Patents

Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen

Info

Publication number
ATE520707T1
ATE520707T1 AT02797050T AT02797050T ATE520707T1 AT E520707 T1 ATE520707 T1 AT E520707T1 AT 02797050 T AT02797050 T AT 02797050T AT 02797050 T AT02797050 T AT 02797050T AT E520707 T1 ATE520707 T1 AT E520707T1
Authority
AT
Austria
Prior art keywords
sequences
aav
adeno
isolation
identification
Prior art date
Application number
AT02797050T
Other languages
English (en)
Inventor
Guangping Gao
James Wilson
Mauricio Alvira
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=ATE520707(T1) "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
Application granted granted Critical
Publication of ATE520707T1 publication Critical patent/ATE520707T1/de

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biotechnology (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)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Rheumatology (AREA)
  • Cell Biology (AREA)
  • Endocrinology (AREA)
AT02797050T 2001-11-13 2002-11-12 Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen ATE520707T1 (de)

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 (en) 2001-11-13 2002-11-12 A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby

Publications (1)

Publication Number Publication Date
ATE520707T1 true ATE520707T1 (de) 2011-09-15

Family

ID=27502639

Family Applications (2)

Application Number Title Priority Date Filing Date
AT02797050T ATE520707T1 (de) 2001-11-13 2002-11-12 Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
AT02257826T ATE317916T1 (de) 2001-11-13 2002-11-12 Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode

Family Applications After (1)

Application Number Title Priority Date Filing Date
AT02257826T ATE317916T1 (de) 2001-11-13 2002-11-12 Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode

Country Status (22)

Country Link
US (15) US20030138772A1 (de)
EP (4) EP2338900B1 (de)
JP (6) JP4677187B2 (de)
KR (2) KR101014207B1 (de)
CN (6) CN103555677B (de)
AT (2) ATE520707T1 (de)
AU (1) AU2002361573B2 (de)
BR (3) BRPI0214119B8 (de)
CA (7) CA2945734C (de)
DE (1) DE60209193T2 (de)
DK (1) DK1310571T3 (de)
ES (3) ES2439515T3 (de)
HU (2) HU230406B1 (de)
IL (11) IL161827A0 (de)
MX (3) MX359371B (de)
NO (4) NO334379B1 (de)
NZ (7) NZ600121A (de)
PH (2) PH12014501487A1 (de)
PL (4) PL220644B1 (de)
SG (5) SG168422A1 (de)
WO (1) WO2003042397A2 (de)
ZA (1) ZA200403360B (de)

Families Citing this family (622)

* 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
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
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
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
US20040121311A1 (en) 2002-12-06 2004-06-24 Ecker David J. Methods for rapid detection and identification of bioagents in livestock
US20030027135A1 (en) 2001-03-02 2003-02-06 Ecker David J. Method for rapid detection and identification of bioagents
US8073627B2 (en) 2001-06-26 2011-12-06 Ibis Biosciences, Inc. System for indentification of pathogens
US7217510B2 (en) 2001-06-26 2007-05-15 Isis Pharmaceuticals, Inc. Methods for providing bacterial bioagent characterizing information
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
JP4810062B2 (ja) * 2001-12-17 2011-11-09 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 アデノ随伴ウイルス（ａａｖ）血清型８の配列
EP1359217B1 (de) 2002-04-29 2006-12-13 The Trustees of The University of Pennsylvania Methode für die direkte Gewinnung und Amplifikation von integrierten Viren aus zellulärer Gewebe-DNA
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
EP1578399A4 (de) 2002-12-06 2007-11-28 Isis Pharmaceuticals Inc Verfahren für die rasche identifikation von pathogenen bei tier und mensch
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
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
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
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
PL2357189T3 (pl) * 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
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
AU2010201278B2 (en) * 2003-09-01 2012-11-15 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
NZ545656A (en) * 2003-09-01 2009-02-28 Amc Amsterdam AAV vectors for in vivo gene therapy of rheumatoid arthritis
US8546082B2 (en) 2003-09-11 2013-10-01 Ibis Biosciences, Inc. Methods for identification of sepsis-causing bacteria
US8242254B2 (en) 2003-09-11 2012-08-14 Ibis Biosciences, Inc. Compositions for use in identification of bacteria
US8097416B2 (en) 2003-09-11 2012-01-17 Ibis Biosciences, Inc. Methods for identification of sepsis-causing bacteria
EP3910063A1 (de) * 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Kladen aus adeno-assoziiertem virus (aav), sequenzen, vektoren damit und verwendungen davon
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 (en) * 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 Bovine adeno-associated viral (baav) vector and uses thereof
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
ES2361000T3 (es) * 2004-04-28 2011-06-13 The Trustees Of The University Of Pennsylvania Suministro secuencial de moléculas inmunogénicas mediante administraciones de un adenovirus y de un virus adeno-asociado.
JP2007535541A (ja) 2004-04-28 2007-12-06 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 Ｅ４欠失アデノウイルス初回免疫およびｅ１欠失アデノウイルス追加免疫を用いる免疫化方式
JP4810533B2 (ja) 2004-05-24 2011-11-09 アイビス バイオサイエンシズ インコーポレイティッド ディジタルスレショルド化による選択的イオン濾過作用を用いた質量分光測定法
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
WO2006073496A2 (en) * 2004-07-30 2006-07-13 Targeted Genetics Corporation Recombinant aav based vaccine methods
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
WO2006135400A2 (en) 2004-08-24 2006-12-21 Isis Pharmaceuticals, Inc. Methods for rapid identification of recombinant organisms
EA200700807A1 (ru) * 2004-10-05 2007-08-31 Мерц Фарма Гмбх Унд Ко. Кгаа Новые циклические и ациклические пропеноны для лечения заболеваний цнс
US8182992B2 (en) 2005-03-03 2012-05-22 Ibis Biosciences, Inc. Compositions for use in identification of adventitious viruses
US8084207B2 (en) 2005-03-03 2011-12-27 Ibis Bioscience, Inc. Compositions for use in identification of papillomavirus
EP3409296A1 (de) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
WO2006119432A2 (en) * 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
WO2007014045A2 (en) 2005-07-21 2007-02-01 Isis Pharmaceuticals, Inc. Methods for rapid identification and quantitation of nucleic acid variants
EP1957678B1 (de) * 2005-11-28 2012-06-13 Ibis Biosciences, Inc. Zusammensetzung zur verwendung bei der identifikation adventiver kontaminationsviren
EP2007795B1 (de) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav-capsid-proteine
JP5268890B2 (ja) 2006-04-28 2013-08-21 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 Ａａｖの規模適応性の製造方法
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 (en) 2006-09-14 2008-11-27 Ibis Biosciences, Inc. Targeted whole genome amplification method for identification of pathogens
JP5680304B2 (ja) 2007-02-23 2015-03-04 アイビス バイオサイエンシズ インコーポレイティッド 迅速な法医学的ｄｎａ分析法
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
EP3492596A1 (de) 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav-vektorzusammensetzungen mit tyrosinmodifizierten kapsidproteinen und verfahren zu deren verwendung
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
WO2008151023A2 (en) 2007-06-01 2008-12-11 Ibis Biosciences, Inc. Methods and compositions for multiple displacement amplification of nucleic acids
EP2058401A1 (de) * 2007-10-05 2009-05-13 Genethon Weitverbreitete Genverabreichung an Motorneuronen unter Verwendung einer peripheren Injektion von AAV-Vektoren
EP2910637A1 (de) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vektoren für die übertragung licht-empfindlicher proteine und deren verwendung
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2349549B1 (de) 2008-09-16 2012-07-18 Ibis Biosciences, Inc. Mischpatronen, mischstationen und verwandte ausrüstungen, und system
WO2010033625A1 (en) 2008-09-16 2010-03-25 Ibis Biosciences, Inc. Microplate handling systems and related computer program products and methods
EP2347254A2 (de) 2008-09-16 2011-07-27 Ibis Biosciences, Inc. Probenverarbeitungseinheiten, -systeme und damit verbundene verfahren
WO2010093943A1 (en) 2009-02-12 2010-08-19 Ibis Biosciences, Inc. Ionization probe assemblies
ES2724122T3 (es) 2009-04-30 2019-09-06 Univ Pennsylvania Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2010143761A1 (ko) * 2009-06-12 2010-12-16 (주)바이오니아 미지시료 내 감염성 미생물을 신속하게 검출하는 방법
WO2011008972A1 (en) 2009-07-17 2011-01-20 Ibis Biosciences, Inc. Systems for bioagent identification
WO2011008971A1 (en) 2009-07-17 2011-01-20 Ibis Biosciences, Inc. Lift and mount apparatus
WO2011041502A1 (en) 2009-10-01 2011-04-07 The Trustees Of The University Of Pennsylvania Aav vectors expressing sec1o for treating kidney damage
US9890408B2 (en) 2009-10-15 2018-02-13 Ibis Biosciences, Inc. Multiple displacement amplification
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
EP3540055A1 (de) 2010-04-23 2019-09-18 University of Massachusetts Auf das zns abzielende aav-vektoren und verfahren zur verwendung davon
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
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
EP2699688A1 (de) 2011-04-20 2014-02-26 The Trustees Of The University Of Pennsylvania Dosierpläne und zusammensetzungen zur aav-vermittelten passiven immunisierung von luftbürtigen krankheitserregern
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
LT3254703T (lt) 2011-04-22 2020-05-25 The Regents Of The University Of California Adenoasocijuoto viruso virionai su variantine kapside ir jų panaudojimo būdai
EP2748185A1 (de) * 2011-08-24 2014-07-02 The Board of Trustees of The Leland Stanford Junior University Neue aav-capsid-proteine zum transport von nukleinsäuren
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
CN104520428B (zh) * 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
WO2013130393A1 (en) * 2012-02-28 2013-09-06 Cornell University Aav-directed persistent expression of an anti-nicotine antibody gene for smoking cessation
WO2013154744A1 (en) * 2012-04-13 2013-10-17 Cornell University Development of a highly efficient second generation nicotine-conjugate vaccine to treat nicotine addiction
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
DK2954051T3 (da) 2013-02-08 2019-07-08 Univ Pennsylvania Modificeret kapsid til genoverførsel til behandling af nethinden
PL2956477T5 (pl) 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Zoptymalizowany gen czynnika 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
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
WO2014194132A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
PH12016500162B1 (en) 2013-07-22 2024-02-21 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues
EP3099333B8 (de) * 2014-01-31 2020-12-30 Temple University of the Commonwealth System of Higher Education Bag3 als target zur behandlung von herzinsuffizienz
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
SI3116900T1 (sl) 2014-03-09 2021-02-26 The Trustees Of The University Of Pennsylvania Sestavki uporabni pri zdravljenju pomanjkanja ornitin transkarbamilaze(OTC)
CA2942776C (en) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter
JP6756700B2 (ja) 2014-03-18 2020-09-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
UA120050C2 (uk) 2014-03-21 2019-09-25 Джензайм Корпорейшн Генна терапія для лікування пігментного ретиніту
EP2933335A1 (de) 2014-04-18 2015-10-21 Genethon Verfahren zur Behandlung peripherer Neuropathien und Motoneuronerkrankungen
WO2015164778A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennysylvania Ldlr variants and their use in compositions for reducing cholesterol levels
JP6983511B2 (ja) 2014-04-25 2021-12-17 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 脳中の転移乳癌および他の癌を処置するための方法および組成物
EP3134522B1 (de) 2014-04-25 2021-10-06 University of Massachusetts Rekombinante aav-vektoren zur reduzierung der immunität gegen transgene produkte
ES2833230T3 (es) 2014-05-13 2021-06-14 Univ Pennsylvania Composiciones que comprenden AVV que expresan construcciones y usos de doble anticuerpo del mismo
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
EP3151866B1 (de) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimäre kapside
EP2960336A1 (de) 2014-06-27 2015-12-30 Genethon Effiziente systemische Behandlung von Muskeldystrophie
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
JP6619758B2 (ja) 2014-07-03 2019-12-11 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム 疾患を処置するためのｇｌｓ１阻害薬
EP4012035B1 (de) 2014-09-16 2024-11-06 Genzyme Corporation Adeno-assoziierte virale vektoren zur behandlung von myocilin (myoc)-glaukom
ES3011359T3 (en) 2014-09-16 2025-04-07 Genzyme Corp Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EP3242945B1 (de) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Einzelvektor-genkonstrukt mit insulin- und glucokinasegenen
MX2017009336A (es) 2015-01-16 2017-11-15 Voyager Therapeutics Inc Polinucleótidos dirigidos al sistema nervioso 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
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
CR20170407A (es) 2015-02-10 2017-11-14 Genzyme Corp Mejora del suministro de partículas virales al cuerpo estriado y al córtex
MA40819B1 (fr) 2015-02-10 2020-04-30 Genzyme Corp Variant d'arni
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
WO2016145217A1 (en) 2015-03-10 2016-09-15 The Trustees Of Columbia University In The City Of New York Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression
WO2016154344A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
US11020443B2 (en) 2015-04-23 2021-06-01 University Of Massachusetts Modulation of AAV vector transgene expression
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
US20180110877A1 (en) 2015-04-27 2018-04-26 The Trustees Of The University Of Pennsylvania DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3307310A2 (de) 2015-05-13 2018-04-18 The Trustees Of The University Of Pennsylvania Aav-vermittelte expression von anti-influenza-antikörpern und verfahren zur verwendung davon
MX2017014665A (es) 2015-05-16 2018-04-24 Genzyme Corp Edicion de genes de mutaciones intronicas profundas.
CA2990193A1 (en) 2015-06-23 2016-12-29 The Children's Hospital Of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
HK1256909A1 (zh) 2015-08-06 2019-10-04 宾夕法尼亚大学 Glp-1和其在用於治疗代谢疾病的组合物中的用途
KR102699944B1 (ko) 2015-08-25 2024-09-13 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
CN108137664B (zh) 2015-08-31 2021-11-26 宾夕法尼亚州大学信托人 用于治疗伴侣动物的aav-epo
AU2016326627B2 (en) 2015-09-24 2022-12-15 The Trustees Of The University Of Pennsylvania Composition and method for treating complement-mediated disease
PT3356390T (pt) * 2015-09-28 2021-04-21 Univ Florida Métodos e composições para vetores virais que se evadem a anticorpos
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
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
DK3364997T5 (da) 2015-10-22 2024-09-30 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
EP3368054A4 (de) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. Regulierbare expression unter verwendung des adeno-assoziierten virus (aav)
EP3368563A1 (de) 2015-10-28 2018-09-05 The Trustees Of The University Of Pennsylvania Intrathekale verabreichung von adeno-assoziierten viralen vektoren zur gentherapie
HK1254836A1 (zh) 2015-10-30 2019-07-26 Nbe-Therapeutics Ag 抗-ror1抗体
US10983110B2 (en) 2015-12-02 2021-04-20 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
EP3387138B1 (de) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Skalierbares reinigungsverfahren für aav9
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387117B1 (de) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Skalierbares reinigungsverfahren für aav8
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
RU2762747C2 (ru) 2015-12-14 2021-12-22 Зе Трастис Оф Зе Юниверсити Оф Пенсильвания Генная терапия офтальмологических нарушений
JP7061067B2 (ja) 2015-12-14 2022-04-27 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 クリグラー・ナジャー症候群の処置のための組成物
CN108603199A (zh) 2015-12-15 2018-09-28 建新公司 用于治疗粘脂贮积症ii型的腺伴随病毒载体
SG11201804664XA (en) 2015-12-22 2018-07-30 Univ Texas Salt forms and polymorphs of (r)-1-(4-(6-(2-(4-(3,3-difluorocyclobutoxy)-6-methylpyridin-2-yl) acetamido) pyridazin-3-yl)-2-fluorobutyl)-n-methyl-1h-1,2,3-triazole-4-carboxamide
BR112018014615A2 (pt) 2016-01-20 2018-12-11 The Scripps Research Institute composições de anticorpo para ror1 e métodos relacionados
PT3411478T (pt) 2016-02-01 2022-09-13 Bioverativ Therapeutics Inc Genes do fator viii otimizados
EP3411059A4 (de) 2016-02-02 2019-10-16 University Of Massachusetts Verfahren zur erhöhung der wirksamkeit der systemischen abgabe von avv-genen an das zentrale nervensystem
EP3411488A1 (de) 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gentherapie zur behandlung von mukopolysaccharidose typ i
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
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 (de) 2016-04-05 2019-11-27 University of Massachusetts Zusammensetzungen und verfahren zur selektiven hemmung der expression des grainyhead-like-proteins
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2017180936A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
IL305449B1 (en) 2016-04-15 2025-09-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
WO2017180861A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsulvania Gene therapy for treating hemophilia b
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
US11091776B2 (en) * 2016-04-15 2021-08-17 The Trustees Of The University Of Pennsylvania AAV8 mutant capsids and compositions containing same
KR102450833B1 (ko) 2016-04-15 2022-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 혈우병 a 치료용 유전자 요법
US11401527B2 (en) 2016-04-17 2022-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
EP3448987A4 (de) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
EP3448874A4 (de) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Zusammensetzungen zur behandlung einer erkrankung
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
MX2018013463A (es) 2016-05-13 2019-07-04 4D Molecular Therapeutics Inc Capsides variantes de virus adenoasociados y metodos de uso de las mismas.
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
WO2017212929A1 (ja) * 2016-06-08 2017-12-14 ソニー株式会社 撮像制御装置および方法、並びに車両
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
MX2019000221A (es) 2016-07-05 2020-02-07 Univ Massachusetts Suministro genetico de sfasl mediado por aav2 como una terapia neuro-protectora en el glaucoma.
CN110191954B (zh) 2016-07-08 2024-05-14 宾夕法尼亚州大学信托人 用于治疗涉及rdh12的病症和疾病的方法和组合物
EP4275747A3 (de) 2016-07-19 2024-01-24 Duke University Therapeutische anwendungen von cpf1-basierter genomeditierung
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
EP3491008A2 (de) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Neuartige adeno-assoziierte virus-kapsid-proteine
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
US11698377B2 (en) 2016-08-15 2023-07-11 Genzyme Corporation Methods for detecting AAV
WO2018035503A1 (en) 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
EP3518985A4 (de) 2016-09-29 2020-08-05 University of Florida Research Foundation, Incorporated Aavrh.10-varianten mit austrittsmöglichkeiten für wirtsantikörper und veränderten gewebe-targeting-eigenschaften
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
EP3528785A4 (de) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. Modifizierte aav-kaspide und verwendungen davon
EP3548065B1 (de) 2016-12-01 2022-11-09 INSERM - Institut National de la Santé et de la Recherche Médicale Pharmazeutische zusammensetzungen zur behandlung von degenerativen netzhauterkrankungen
WO2018112225A1 (en) * 2016-12-14 2018-06-21 The J. David Gladstone Institutes Methods and compositions for generating a deletion library and for identifying a defective interfering particle (dip)
MX2019007876A (es) * 2016-12-30 2019-10-15 Univ Pennsylvania Terapia genetica para tratar fenilcetonuria.
WO2018136710A1 (en) 2017-01-20 2018-07-26 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of krabbe disease with umbilical cord blood transplantion (ucbt) and increased galactocerebrosidase (galc) expression
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11827906B2 (en) 2017-02-28 2023-11-28 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
AU2018229293A1 (en) 2017-02-28 2019-08-29 Janssen Biotech, Inc. Influenza vaccines based on AAV vectors
KR20230093072A (ko) 2017-03-01 2023-06-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 안구 장애에 대한 유전자 치료
US11376321B2 (en) 2017-03-02 2022-07-05 Genethon Method for removing anti-AAV antibodies from a blood-derived composition
EP3606544B1 (de) 2017-04-05 2025-06-04 University of Massachusetts Minigentherapie
WO2018189208A1 (en) 2017-04-10 2018-10-18 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
US11613739B2 (en) 2017-04-14 2023-03-28 Regenxbio Inc. Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
EP3634986A4 (de) 2017-04-24 2021-09-08 The Trustees of The University of Pennsylvania Gentherapie für augenerkrankungen
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
CA3061655A1 (en) 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
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
AU2018265541B2 (en) 2017-05-12 2023-05-25 University Of Massachusetts Viral vector production
EP3630986A4 (de) 2017-05-31 2021-08-11 The Trustees of The University of Pennsylvania Gentherapie zur behandlung von peroxisomalen erkrankungen
AU2018281145A1 (en) 2017-06-06 2020-01-02 University Of Massachusetts Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome
JP7766393B2 (ja) 2017-06-14 2025-11-10 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
US10693918B2 (en) 2017-06-15 2020-06-23 Palo Alto Networks, Inc. Radio access technology based security in service provider networks
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
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
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
US10812532B2 (en) 2017-06-15 2020-10-20 Palo Alto Networks, Inc. Security for cellular internet of things in mobile networks
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11050789B2 (en) 2017-06-15 2021-06-29 Palo Alto Networks, Inc. Location based security in service provider networks
AU2018288863A1 (en) * 2017-06-22 2020-01-30 Board Of Regents, The University Of Texas System Methods for producing regulatory immune cells and uses thereof
CN110461368A (zh) 2017-06-30 2019-11-15 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及其使用方法
AU2018298133A1 (en) 2017-07-06 2020-01-23 The Trustees Of The University Of Pennsylvania AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
WO2019008157A1 (en) 2017-07-07 2019-01-10 Genethon NOVEL POLYNUCLEOTIDES ENCODING HUMAN FKRP PROTEIN
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3662060A2 (de) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur aav-freisetzung
SG11202000846WA (en) 2017-08-07 2020-02-27 Nbe Therapeutics Ag Anthracycline-based antibody drug conjugates having high in vivo tolerability
EP3676373A4 (de) * 2017-08-28 2021-06-09 The Regents of The University of California Capsid-varianten von adeno-assoziierten viren und verfahren zur verwendung davon
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
SG11202002457RA (en) 2017-09-22 2020-04-29 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
WO2019060686A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF
WO2019060726A1 (en) 2017-09-22 2019-03-28 Genzyme Corporation Variant rnai
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
AU2018346102B2 (en) * 2017-10-03 2023-05-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US12441998B2 (en) 2017-10-12 2025-10-14 The Trustees Of Columbia University In The City Of New York SLC2A1 lncRNA as a biologic and related treatments and methods
EP4454654A3 (de) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Behandlung von amyotropher lateralsklerose (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2019079496A2 (en) 2017-10-18 2019-04-25 Regenxbio, Inc. FULLY HUMAN ANTIBODY-BASED THERAPEUTIC AGENTS HAVING POST-TRANSLATION MODIFICATION
EP3697764B1 (de) * 2017-10-18 2025-06-11 Board Of Regents, The University Of Texas System Gls-1-inhibitoren zur verwendung bei der behandlung von krebs
EP3697449A1 (de) 2017-10-18 2020-08-26 REGENXBIO Inc. Behandlung von augenkrankheiten und metastasierendem dickdarmkrebs mit menschlicher post-translational modifizierter vegf-falle
JP7184894B2 (ja) 2017-11-27 2022-12-06 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ随伴ウイルス変異キャプシドおよび血管新生の阻害のための使用
KR20200104864A (ko) 2017-11-30 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 뮤코다당류증 iiib형에 대한 유전자 요법
JP7389744B2 (ja) 2017-11-30 2023-11-30 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 ムコ多糖症ｉｉｉａ型のための遺伝子療法
MX2020006435A (es) 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
WO2019152843A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
SG11202007942YA (en) 2018-02-27 2020-09-29 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
US20210010028A1 (en) 2018-03-06 2021-01-14 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
KR20200135433A (ko) 2018-03-23 2020-12-02 유니버시티 오브 매사추세츠 골 장애 치료를 위한 유전자 치료제
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，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
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
JP7575273B2 (ja) 2018-04-16 2024-10-29 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 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法
US11981892B2 (en) 2018-04-16 2024-05-14 University Of Massachusetts Compositions and methods for improved gene editing
US11472848B2 (en) 2018-04-27 2022-10-18 University Of Massachusetts AAV capsids identified by in vivo library selection
US12378576B2 (en) 2018-04-27 2025-08-05 Spacecraft Seven, Llc Gene therapy for CNS degeneration
US12365865B2 (en) 2018-04-29 2025-07-22 Regenxbio Inc. Scalable clarification process for recombinant AAV production
WO2019212922A1 (en) 2018-04-29 2019-11-07 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
CA3098871A1 (en) 2018-05-08 2019-11-14 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
EP3790627A2 (de) 2018-05-09 2021-03-17 BioMarin Pharmaceutical Inc. Verfahren zur behandlung von phenylketonurie
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
JP2021523914A (ja) 2018-05-15 2021-09-09 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． パーキンソン病を治療するための組成物および方法
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒（ａａｖ）之組成物和方法
EP3793686A1 (de) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Aav-serotypen zur gehirnspezifischen payload-übertragung
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
EP3806888B1 (de) 2018-06-12 2024-01-31 Obsidian Therapeutics, Inc. Von pde5 abgeleitete regulatorische konstrukte und verfahren zur verwendung in der immuntherapie
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
US12070702B2 (en) 2018-06-14 2024-08-27 Regenxbio Inc. Anion exchange chromatography for recombinant AAV production
JP7565218B2 (ja) 2018-07-02 2024-10-10 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
MX2021000443A (es) 2018-07-12 2021-05-28 Spacecraft Seven Llc Vectores de terapia génica para el tratamiento de la enfermedad de danon.
SG11202100139VA (en) 2018-07-17 2021-02-25 Neuromyon Inc Treatment of neuropathy with dna constructs expressing igf-1 isoforms
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US12188040B2 (en) * 2018-07-30 2025-01-07 Gene Therapy Research Institution Co., Ltd. Method for enhancing gene expression using AAV vector
WO2020028816A1 (en) 2018-08-03 2020-02-06 Genzyme Corporation Variant rnai against alpha-synuclein
AU2019319976B2 (en) 2018-08-10 2025-08-14 Regenxbio Inc. Scalable method for recombinant AAV production
SG11202102058UA (en) 2018-08-30 2021-03-30 Tenaya Therapeutics Inc Cardiac cell reprogramming with myocardin and ascl1
US12274733B2 (en) 2018-09-28 2025-04-15 President And Fellows Of Harvard College Cellular reprogramming to reverse aging and promote organ and tissue regeneration
AU2019347666B2 (en) 2018-09-28 2025-10-02 President And Fellows Of Harvard College Cellular reprogramming to reverse aging and promote organ and tissue regeneration
CA3113470A1 (en) 2018-09-28 2020-04-02 President And Fellows Of Harvard College Mutant reverse tetracycline transactivators for expression of genes
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
PE20211596A1 (es) 2018-10-01 2021-08-18 Univ Pennsylvania Composiciones utiles para tratar la gangliosidosis gm1
AR116569A1 (es) 2018-10-01 2021-05-19 Ultragenyx Pharmaceutical Inc Terapia génica para tratar la acidemia propiónica
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
MX2021004213A (es) 2018-10-12 2021-07-16 Genzyme Corp Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado.
WO2020081415A1 (en) 2018-10-15 2020-04-23 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
EP3867389A1 (de) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expressionsvektoren zur massenproduktion von raav im baculovirus/sf9-system
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
EP3880823A4 (de) * 2018-11-16 2022-08-17 Asklepios Biopharmaceutical, Inc. Therapeutisches adeno-assoziiertes virus zur behandlung von morbus pompe
CN113271982B (zh) 2018-11-16 2024-07-30 摩大力斯医疗株式会社 通过靶向肌营养相关蛋白基因来治疗肌营养不良的方法
US12391947B2 (en) 2018-11-29 2025-08-19 University Of Massachusetts Modulation of SPTLC1 via recombinant adeno-associated vectors
WO2020140007A1 (en) 2018-12-28 2020-07-02 University Of Rochester Gene therapy for best1 dominant mutations
US12338450B2 (en) 2019-01-04 2025-06-24 Ultragenyx Pharmaceutical Inc. Gene therapy constructs for treating Wilson disease
US12371698B2 (en) 2019-01-14 2025-07-29 University Of Rochester Targeted nuclear RNA cleavage and polyadenylation with CRISPR-Cas
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
KR20210130158A (ko) 2019-01-31 2021-10-29 오레곤 헬스 앤드 사이언스 유니버시티 Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
US20220054655A1 (en) 2019-02-22 2022-02-24 University Of Massachusetts Oxr1 gene therapy
KR20210131370A (ko) 2019-02-22 2021-11-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
EP3931334A1 (de) 2019-02-25 2022-01-05 Novartis AG Zusammensetzungen und verfahren zur behandlung von bietti-kristall-dystrophie
JP2022520875A (ja) 2019-02-25 2022-04-01 ノバルティス アーゲー Ｂｉｅｔｔｉクリスタリン網膜症を治療するための組成物及び方法
AU2020228028A1 (en) 2019-02-25 2021-09-30 University Of Massachusetts DNA-binding domain transactivators and uses thereof
PE20212072A1 (es) 2019-02-26 2021-10-26 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe
US20220047618A1 (en) 2019-02-28 2022-02-17 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
AU2020241888A1 (en) 2019-03-21 2021-09-30 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors
EP3947427A1 (de) 2019-03-25 2022-02-09 Genethon Herstellung von grossen quasidystrophinen mit überlappenden aav-vektoren
CA3135609A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP4667578A2 (de) 2019-04-03 2025-12-24 REGENXBIO Inc. Gentherapie für augenerkrankungen
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
HUE064411T2 (hu) 2019-04-11 2024-03-28 Regenxbio Inc Méretkizárásos kromatográfiás módszerek rekombináns adeno-asszociált víruskészítmények jellemzésére
BR112021020668A2 (pt) 2019-04-17 2022-01-11 Codiak Biosciences Inc Composições de exossomos e aav
CN113993552A (zh) 2019-04-19 2022-01-28 再生生物股份有限公司 腺相关病毒载体制剂和方法
SG11202111414RA (en) 2019-04-24 2021-11-29 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
WO2020219990A1 (en) 2019-04-26 2020-10-29 President And Fellows Of Harvard College Aav vectors encoding mini-pcdh15 and uses thereof
WO2020223236A1 (en) * 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
EP3962536A1 (de) 2019-04-29 2022-03-09 Voyager Therapeutics, Inc. Systeme und verfahren zur herstellung von baculovirusinfizierten insektenzellen (biics) in bioreaktoren
BR112021021908A2 (pt) 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3972610B1 (de) 2019-05-20 2025-09-03 University of Massachusetts Minigentherapie
KR20220011664A (ko) * 2019-05-24 2022-01-28 리제너론 파마슈티칼스 인코포레이티드 변형된 바이러스 입자 및 이의 용도
JP7565620B2 (ja) 2019-05-28 2024-10-11 株式会社モダリス Ｄｍｐｋ遺伝子を標的とした筋ジストロフィーの治療方法
EP3987024A4 (de) 2019-06-20 2023-11-01 University Of Massachusetts Zusammensetzungen und verfahren zur verbesserten geneditierung
BR112021026866A2 (pt) 2019-07-02 2022-03-03 M6P Therapeutics Composições de vetor e métodos de seu uso para o tratamento de distúrbios do armazenamento lisossômico
WO2021007473A1 (en) 2019-07-10 2021-01-14 Masonic Medical Research Institute Vgll4 with ucp-1 cis-regulatory element and method of use thereof
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
EP3997226A1 (de) 2019-07-11 2022-05-18 Tenaya Therapeutics, Inc. Umprogrammierung von herzzellen mit mikrornas und anderen faktoren
AU2020311618B2 (en) 2019-07-11 2025-04-10 Centre Hospitalier Universitaire De Nantes Chemically-modified adeno-associated virus
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
WO2021016453A1 (en) 2019-07-23 2021-01-28 University Of Rochester Targeted rna cleavage with crispr-cas
WO2021021661A1 (en) 2019-07-26 2021-02-04 Regenxbio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
EP4007814A1 (de) 2019-07-26 2022-06-08 Akouos, Inc. Verfahren zur behandlung von hörverlust unter verwendung eines sekretierten zielproteins
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4013770A4 (de) * 2019-08-14 2023-12-13 University of Florida Research Foundation, Incorporated Aav-kapsidvarianten für die gentherapie
TW202122582A (zh) 2019-08-26 2021-06-16 美商航海家醫療公司 病毒蛋白之控制表現
MX2022002366A (es) 2019-08-26 2022-07-19 Regenxbio Inc Tratamiento de la retinopatía diabética con fab anti-vegf completamente humano modificado postraduccionalmente.
EP4022057A1 (de) 2019-08-27 2022-07-06 Vertex Pharmaceuticals Incorporated Zusammensetzungen und verfahren zur behandlung von erkrankungen im zusammenhang mit repetitiver dna
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021046451A1 (en) 2019-09-06 2021-03-11 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
CN114521143B (zh) 2019-09-19 2025-03-14 吉尼松公司 减轻fkrp心脏毒性的基因治疗表达系统
US11492622B2 (en) 2019-09-26 2022-11-08 Massachusetts Institute Of Technology MicroRNA-based logic gates and uses thereof
WO2021062092A1 (en) 2019-09-26 2021-04-01 Massachusetts Institute Of Technology Trans-activated functional rna by strand displacement and uses thereof
WO2021067389A1 (en) 2019-09-30 2021-04-08 Bioverativ Therapeutics Inc. Lentiviral vector formulations
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
KR20220081365A (ko) 2019-10-07 2022-06-15 리젠엑스바이오 인크. 아데노-연관 바이러스 벡터 약제학적 조성물 및 방법
WO2021076656A1 (en) 2019-10-15 2021-04-22 University Of Massachusetts Rna editor-enhanced rna trans-splicing
WO2021073568A1 (en) * 2019-10-16 2021-04-22 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
AU2020384294A1 (en) 2019-11-14 2022-06-02 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with liver-specific gene therapy vectors
US20230016983A1 (en) 2019-11-19 2023-01-19 lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) Antisense oligonucleotides and thier use for the treatment of cancer
JP2023503637A (ja) * 2019-11-28 2023-01-31 リジェネックスバイオ インコーポレイテッド マイクロジストロフィン遺伝子治療コンストラクト及びその使用
EP4072595A1 (de) 2019-12-10 2022-10-19 Takeda Pharmaceutical Company Limited Adeno-assoziierte virusvektoren zur behandlung der hunter-krankheit
KR20220128632A (ko) 2019-12-31 2022-09-21 스완바이오 테라퓨틱스 리미티드 개선된 aav-abcd1 구축물 및 부신백질이영양증 (ald) 및/또는 부신척수신경병증 (amn)의 치료 또는 예방을 위한 용도
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
WO2021150570A1 (en) 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
JP2023512043A (ja) 2020-01-29 2023-03-23 レジェンクスバイオ インコーポレーテッド ムコ多糖症ｉｖａの治療
AU2021214174A1 (en) 2020-01-29 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
CA3165713A1 (en) 2020-01-30 2021-08-05 Andrew Scharenberg Bispecific transduction enhancer
CA3165057A1 (en) 2020-02-02 2021-08-05 James M. Wilson Compositions useful for treating gm1 gangliosidosis
EP4100533A1 (de) 2020-02-07 2022-12-14 University of Rochester Ribozym-vermittelte rna-anordnung und expression
US20230078498A1 (en) 2020-02-07 2023-03-16 University Of Rochester Targeted Translation of RNA with CRISPR-Cas13 to Enhance Protein Synthesis
BR112022015921A2 (pt) 2020-02-14 2022-10-04 Ultragenyx Pharmaceutical Inc Terapia gênica para tratar o transtorno de deficiência de cdkl5
EP4110931A4 (de) 2020-02-25 2024-03-27 University of Massachusetts Induzierbares single-aav-system und verwendungen davon
JP2023518327A (ja) * 2020-02-25 2023-04-28 チルドレンズ・メディカル・リサーチ・インスティテュート アデノ随伴ウイルスカプシドポリペプチドおよびベクター
EP4114421A1 (de) 2020-03-02 2023-01-11 Tenaya Therapeutics, Inc. Genvektorsteuerung durch kardiomyozyten-exprimierte mikrornas
KR20220160069A (ko) 2020-03-27 2022-12-05 유씨비 바이오파마 에스알엘 자율적 노브 도메인 펩티드
JP2023519344A (ja) 2020-03-27 2023-05-10 ユニバーシティ オブ ロチェスター ＣＲＩＳＰＲ－Ｃａｓ１３ ｃｒＲＮＡアレイ
EP4127169A1 (de) 2020-03-27 2023-02-08 University of Rochester Gezielte zerstörung von viraler rna durch crispr-cas13
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
AU2021248577A1 (en) 2020-03-31 2022-09-29 Sichuan Univeristy AAV capsids variants and uses thereof
AR122409A1 (es) 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
KR20220167324A (ko) 2020-04-10 2022-12-20 솔라 바이오사이언시즈 엘엘씨 단백질 응집 장애의 치료를 위한 조성물 및 방법
WO2021211753A1 (en) 2020-04-15 2021-10-21 Voyager Therapeutics, Inc. Tau binding compounds
WO2021222168A2 (en) 2020-04-28 2021-11-04 Sola Biosciences Llc Compositions and methods for the treatment of tdp-43 proteinopathies
CN115803064A (zh) 2020-05-12 2023-03-14 宾夕法尼亚州大学信托人 用于drg特异性降低转基因表达的组合物
AU2021271635A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
EP4165194A2 (de) 2020-05-13 2023-04-19 Akouos, Inc. Zusammensetzungen und verfahren zur behandlung von kcnq4-assoziiertem gehörverlust
CA3183171A1 (en) 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating slc26a4-associated hearing loss
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
JP2023529371A (ja) 2020-06-05 2023-07-10 ソラ・バイオサイエンシズ・エルエルシー シヌクレイノパチーの処置のための組成物および方法
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
JP2023530171A (ja) 2020-06-19 2023-07-13 ジェネトン 筋肉内及び心臓内でのｓｇｃｇの適切な発現を可能にする遺伝子療法発現系
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
JP2023532806A (ja) 2020-07-10 2023-07-31 ジェネトン 新規の筋肉特異的プロモーター
EP4179097A1 (de) 2020-07-13 2023-05-17 The Trustees of The University of Pennsylvania Zusammensetzungen zur behandlung von morbus charcot-marie-tooth
EP4182455A1 (de) 2020-07-15 2023-05-24 University of Rochester Gezielte rna-spaltung mit dcosl3-rnase-fusionsproteinen
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4189095A1 (de) 2020-07-27 2023-06-07 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von neurologischen erkrankungen im zusammenhang mit glucosylceramidase-beta-mangel
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
CN116209430A (zh) 2020-08-07 2023-06-02 阿米库斯治疗学公司 囊泡靶向蛋白及其用途
CA3185267A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
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
AU2021325954A1 (en) 2020-08-14 2023-03-02 The Trustees Of The University Of Pennsylvania Novel AAV capsids and compositions containing same
AR123288A1 (es) 2020-08-19 2022-11-16 Stridebio Inc Vectores de virus adenoasociados para el tratamiento del síndrome de rett
GB202013194D0 (en) 2020-08-24 2020-10-07 Combigene Ab Gene therapy for lipodystrophy
EP4200429A1 (de) 2020-08-24 2023-06-28 The Trustees of The University of Pennsylvania Virale vektoren zur codierung von glp-1-rezeptoragonistenfusionen und verwendungen davon bei der behandlung von stoffwechselerkrankungen
JP2023540054A (ja) 2020-08-26 2023-09-21 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 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
TW202218686A (zh) 2020-09-09 2022-05-16 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)之組合物及方法
CN116323641A (zh) 2020-09-10 2023-06-23 路德维希-马克西米利安慕尼黑大学 工程化的aav载体
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
EP4213890A1 (de) 2020-09-15 2023-07-26 RegenxBio Inc. Vektorisierte lanadlumab und verabreichung davon
EP4214242A1 (de) 2020-09-15 2023-07-26 RegenxBio Inc. Vektorisierte antikörper für antivirale therapie
US20230383278A1 (en) 2020-09-18 2023-11-30 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
WO2022066849A1 (en) 2020-09-24 2022-03-31 University Of Massachusetts Aav vectors encoding nf1 and uses thereof
BR112023003929A2 (pt) 2020-10-01 2023-04-11 Genzyme Corp Cassete de expressão de pah humano para tratamento de pku por terapia de reposição gênica direcionada ao fígado
JP2023545424A (ja) 2020-10-07 2023-10-30 リジェネックスバイオ インコーポレイテッド ゲル製剤などの脈絡膜上投与用製剤
IL301643A (en) 2020-10-07 2023-05-01 Regenxbio Inc Gene therapy for ocular manifestations of CLN2 disease
MX2023004035A (es) 2020-10-07 2023-07-05 Regenxbio Inc Formulaciones para la administración supracoroidea tales como formulaciones de viscosidad alta.
JP2023545722A (ja) 2020-10-07 2023-10-31 リジェネックスバイオ インコーポレイテッド 遺伝子治療剤の眼送達のためのアデノ随伴ウイルス
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
CA3198368A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
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
US20230383313A1 (en) 2020-10-18 2023-11-30 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
AU2021371307A1 (en) 2020-10-28 2023-06-01 Regenxbio, Inc. VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
CN116457373A (zh) 2020-10-29 2023-07-18 再生生物股份有限公司 用于眼部适应症的载体化TNF-α拮抗剂
EP4240854A1 (de) 2020-11-06 2023-09-13 Vertex Pharmaceuticals Incorporated Zusammensetzungen und verfahren zur behandlung von dm1 mit sclucas9 und 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
MX2023006444A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen.
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
CR20230228A (es) 2020-12-01 2023-07-18 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
IL303614A (en) 2020-12-16 2023-08-01 Regenxbio Inc Method of producing a recombinant adeno-associated virus particle
EP4271419A1 (de) 2020-12-29 2023-11-08 Akouos, Inc. Zusammensetzungen und verfahren zur behandlung von clrn1-assoziiertem hörverlust und/oder sehverlust
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
CN116981697A (zh) 2021-01-14 2023-10-31 森迪生物科学公司 可分泌有效载荷调节
EP4281568A1 (de) 2021-01-21 2023-11-29 RegenxBio Inc. Verbesserte herstellung von rekombinanten polypeptiden und viren
EP4281559A4 (de) 2021-01-22 2024-12-04 University of Massachusetts Verwendung von neuen mirna-bindungsstellenkassetten zur detargetierung antigenpräsentierender zellen durch vektoren der raav-gentherapie
AU2022212952A1 (en) 2021-01-27 2023-08-10 Umoja Biopharma, Inc. Lentivirus for generating cells expressing anti-cd19 chimeric antigen receptor
AU2022214429A1 (en) 2021-02-01 2023-09-14 Tern Therapeutics, Llc Gene therapy for neuronal ceroid lipofuscinoses
WO2022173605A2 (en) 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
GB202101958D0 (en) 2021-02-12 2021-03-31 Ucl Business Ltd Gene therapy for dopamine transporter deficiency syndrome
KR20230150836A (ko) 2021-02-26 2023-10-31 다케다 야쿠힌 고교 가부시키가이샤 파브리병 치료를 위한 조성물 및 방법
WO2022182959A1 (en) 2021-02-26 2022-09-01 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/slucas9
WO2022182957A1 (en) 2021-02-26 2022-09-01 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/sacas9
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
EP4314295A1 (de) 2021-03-26 2024-02-07 The Board Of Regents Of The University Of Texas System Nukleotidbearbeitung zur neurahmung von dmd-transkripten durch basisbearbeitung und prime-bearbeitung
KR20230170022A (ko) 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
AU2022261125A1 (en) 2021-04-23 2023-11-23 University Of Rochester Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
WO2022229851A1 (en) 2021-04-26 2022-11-03 Crispr Therapeutics Ag Compositions and methods for using slucas9 scaffold sequences
JP2024515974A (ja) 2021-04-26 2024-04-11 ユニバーシティ オブ マサチューセッツ シュタルガルト病（ａｂｃａ４）の遺伝子治療
TW202309293A (zh) 2021-04-26 2023-03-01 美商銳進科斯生物股份有限公司 用於治療肌縮蛋白症(dystrophinopathy)之微小肌縮蛋白基因療法給藥
US20240218397A1 (en) 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
WO2022234519A1 (en) 2021-05-05 2022-11-10 Crispr Therapeutics Ag Compositions and methods for using sacas9 scaffold sequences
EP4337267A1 (de) 2021-05-11 2024-03-20 RegenxBio Inc. Behandlung von duchenne-muskeldystrophie und kombinationen davon
WO2022272296A2 (en) 2021-06-25 2022-12-29 Homology Medicines, Inc. Adeno-associated virus packaging systems
US20240318157A1 (en) 2021-07-01 2024-09-26 Amicus Therapeutics, Inc. Neurotensin Variants And Tagged Proteins Comprising Neurotensin Or Sortilin Propeptide
WO2023278811A1 (en) 2021-07-01 2023-01-05 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
WO2023283649A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
EP4373947A1 (de) 2021-07-19 2024-05-29 New York University Auf1-kombinationstherapien zur behandlung von muskeldegenerativen erkrankungen
EP4377459A2 (de) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Zusammensetzungen und verfahren zur modulation der expression von frataxin (fxn)
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
WO2023018637A1 (en) 2021-08-09 2023-02-16 Vertex Pharmaceuticals Incorporated Gene editing of regulatory elements
US20240425820A1 (en) 2021-08-11 2024-12-26 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
EP4384193A2 (de) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetisch modifizierte zellen für allogene zelltherapie zur reduzierung von durch instant-blut vermittelten entzündungsreaktionen
AU2022325232A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified primary cells for allogeneic cell therapy
JP2024534772A (ja) 2021-08-11 2024-09-26 サナ バイオテクノロジー，インコーポレイテッド 同種異系細胞療法用の遺伝子改変細胞
EP4396237A4 (de) 2021-08-31 2025-11-19 Scout Bio Inc Antigenbindende moleküle und verwendungen davon
EP4399302A2 (de) 2021-09-08 2024-07-17 Vertex Pharmaceuticals Incorporated Präzise exzisionen von teilen von exon51 zur behandlung von duchenne-muskeldystrophie
PE20241065A1 (es) 2021-09-30 2024-05-13 Akouos Inc Composiciones y metodos para el tratamiento de la perdida de audicion asociada a kcnq4
US20240408240A1 (en) 2021-10-01 2024-12-12 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
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
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
EP4413019A2 (de) 2021-10-07 2024-08-14 RegenxBio Inc. Rekombinante adeno-assoziierte viren zur zns-tropen verabreichung
US20250001006A1 (en) 2021-10-07 2025-01-02 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
EP4413024A2 (de) 2021-10-08 2024-08-14 SOLA Biosciences LLC Zusammensetzungen und verfahren zur behandlung von proteopathien
JP2024537178A (ja) 2021-10-08 2024-10-10 ソラ・バイオサイエンシズ・エルエルシー ｐ５３媒介性癌の処置のための組成物および方法
JP2024540987A (ja) 2021-10-21 2024-11-06 バーテックス ファーマシューティカルズ インコーポレイテッド 低免疫細胞
EP4423285A1 (de) 2021-10-28 2024-09-04 RegenxBio Inc. Manipulierte regulatorische nukleinsäureelemente sowie verfahren und verwendungen davon
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20250011812A1 (en) 2021-11-23 2025-01-09 University Of Massachusetts Gene therapy for spinal muscular atrophy
WO2023102442A1 (en) 2021-11-30 2023-06-08 Jaguar Gene Therapy, Llc Gene therapy methods for treatment of diabetes
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
KR20240126864A (ko) 2021-12-17 2024-08-21 타팔지 세러퓨틱스 통증 치료에서 사용을 위한 펩티드 및 방법
EP4442698A4 (de) 2021-12-28 2025-10-15 Chengdu Origen Biotechnology Co Ltd Modifiziertes aav-kapsidprotein und verwendung davon
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
US20250084129A1 (en) * 2022-01-10 2025-03-13 University Of Florida Research Foundation, Incorporated Aav5 capsid variants
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
EP4215614A1 (de) 2022-01-24 2023-07-26 Dynacure Kombinationstherapie für dystrophin-verwandte krankheiten
EP4469584A1 (de) 2022-01-25 2024-12-04 The Trustees of The University of Pennsylvania Aav-kapside zur verbesserten herztransduktion und detargeting der leber
WO2023150142A1 (en) 2022-02-02 2023-08-10 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023154693A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023156530A1 (en) 2022-02-17 2023-08-24 Lysogene Gene therapy for neurodegenerative diseases
IL315000A (en) 2022-02-17 2024-10-01 Sana Biotechnology Inc Engineered cd47 proteins and uses thereof
EP4490291A1 (de) 2022-03-08 2025-01-15 Vertex Pharmaceuticals Incorporated Präzise exzisionen von exonteilen zur behandlung von duchenne-muskeldystrophie
EP4490292A1 (de) 2022-03-08 2025-01-15 Vertex Pharmaceuticals Incorporated Präzise exzisionen von teilen von exon-44, 50 und 53 zur behandlung von duchenne-muskeldystrophie
US20250101382A1 (en) 2022-03-11 2025-03-27 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
EP4493226A1 (de) 2022-03-13 2025-01-22 RegenxBio Inc. Modifizierte muskelspezifische promotoren
US20250213727A1 (en) 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
EP4504950A1 (de) 2022-04-01 2025-02-12 Takeda Pharmaceutical Company Limited Gentherapie für erkrankungen mit zns-manifestationen
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
KR20250007064A (ko) 2022-04-06 2025-01-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Her2 양성 전이성 유방암 및 기타 암을 치료하기 위한 조성물 및 방법
JP2025511883A (ja) 2022-04-06 2025-04-16 プレジデント アンド フェローズ オブ ハーバード カレッジ 中枢神経系の加齢を逆行させること
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
CN119234040A (zh) 2022-04-06 2024-12-31 建新公司 Dm-1肌强直性营养不良的靶向基因疗法
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
WO2023196873A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene forsuprachoidal administration
EP4508222A1 (de) 2022-04-11 2025-02-19 Centre National de la Recherche Scientifique Chemisch modifizierte adeno-assoziierte viren
EP4508062A1 (de) 2022-04-11 2025-02-19 Tenaya Therapeutics, Inc. Adeno-assoziiertes virus mit manipuliertem kapsid
CN119365208A (zh) 2022-04-13 2025-01-24 巴塞罗那自治大学 通过表达klotho蛋白的基因疗法治疗神经肌肉疾病
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250249127A1 (en) 2022-04-14 2025-08-07 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023205606A1 (en) 2022-04-18 2023-10-26 Vertex Pharmaceuticals Incorporated Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver
EP4518973A1 (de) 2022-05-03 2025-03-12 RegenxBio Inc. Vektorisierte anti-tnf-alpha-inhibitoren für augenindikationen
WO2023215806A2 (en) 2022-05-03 2023-11-09 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
AR129441A1 (es) 2022-05-25 2024-08-28 Tafalgie Therapeutics Péptidos y métodos para el tratamiento del dolor
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
CN119731321A (zh) 2022-06-24 2025-03-28 图恩疗法股份有限公司 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法
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
CA3259902A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
CA3259982A1 (en) 2022-06-30 2024-01-04 Indapta Therapeutics, Inc. COMBINATION OF MODIFIED NATURAL KILLER (NK) CELLS AND ANTIBODY THERAPY AND ASSOCIATED METHODS
TW202424201A (zh) 2022-07-06 2024-06-16 美商航海家醫療公司 Ａａｖ殼體變異體及其用途
EP4554967A2 (de) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Zusammensetzungen, systeme und verfahren zur gezielten transkriptionsaktivierung
EP4558634A1 (de) 2022-07-18 2025-05-28 Vertex Pharmaceuticals Incorporated Tandem-guide-rnas (tg-rnas) und deren verwendung bei der genomeditierung
EP4558149A1 (de) 2022-07-21 2025-05-28 Institut National de la Santé et de la Recherche Médicale Verfahren und zusammensetzungen zur behandlung chronischer schmerzerkrankungen
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
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
JP2025527658A (ja) 2022-08-24 2025-08-22 リジェネックスバイオ インコーポレイテッド 組換えアデノ随伴ウイルス及びその使用
JP2025529884A (ja) 2022-08-25 2025-09-09 武田薬品工業株式会社 ファブリー病の治療に使用するための組成物
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
UY40442A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav
CA3267804A1 (en) 2022-09-22 2024-03-28 Dinaqor Ag TREATMENT OF CARDIOMYOPATHY USING AAV GENE THERAPY VECTORS
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB
JP2025534666A (ja) 2022-10-11 2025-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
EP4634394A2 (de) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Rekombinante aav-mutantenvektoren mit herz- und skelettmuskelspezifischen zielmotiven und zusammensetzungen damit
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
US12252518B2 (en) 2023-01-06 2025-03-18 Life Biosciences, Inc. Methods of treating non-arteritic anterior ischemic optic neuropathy
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
WO2024149844A1 (en) 2023-01-12 2024-07-18 Nantes Université Chemically-modified adeno-associated virus
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
AU2024215852A1 (en) 2023-02-03 2025-09-18 Janssen Pharmaceutica Nv Gene therapy vectors for use in parkinson's disease
GB202302480D0 (en) 2023-02-22 2023-04-05 Drishti Discoveries Ltd shRNA for the treatment of disease
CN116286988A (zh) * 2023-03-03 2023-06-23 西咸新区沣厚原创医药科技有限公司 一种稳定表达冠状病毒3cl蛋白酶的腺相关病毒体系、动物模型及构建方法和应用
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024196855A2 (en) 2023-03-17 2024-09-26 University Of Rochester Ribozyme-mediated rna assembly and expression
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
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
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
AR132545A1 (es) 2023-04-26 2025-07-16 Sanofi Sa Líneas celulares hospedadoras y métodos para identificar y usar dichas líneas celulares hospedadoras
WO2024228938A1 (en) * 2023-05-01 2024-11-07 St. Jude Children's Research Hospital, Inc. Modified aav p5 promoter for improved vector titer and purity
WO2024229309A2 (en) 2023-05-03 2024-11-07 Manifold Biotechnologies, Inc. Methodsand compositions for high-throughput protein delivery, screening, and detection
WO2024229302A1 (en) 2023-05-03 2024-11-07 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024233791A1 (en) 2023-05-11 2024-11-14 Seelos Therapeutics, Inc. Methods of treating neurodegenerative disorders
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024243236A2 (en) 2023-05-22 2024-11-28 Sana Biotechnology, Inc. Methods of delivery of islet cells and related methods
WO2024241176A1 (en) 2023-05-24 2024-11-28 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of diseases with central nervous system disorders
WO2024243563A1 (en) 2023-05-25 2024-11-28 Juno Therapeutics, Inc. Aav purification method
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
WO2025017168A1 (en) 2023-07-19 2025-01-23 Genethon Novel optimized utrophin micro-genes
WO2025017169A1 (en) 2023-07-20 2025-01-23 Genethon Novel mididystrophins
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025038430A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025072604A1 (en) 2023-09-28 2025-04-03 University Of Rochester Rna-editing gene therapy approaches for treating myotonic dystrophy type 1 (dm1)
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025080780A1 (en) 2023-10-10 2025-04-17 University Of Rochester Delivery and expression of prime editing crispr systems
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
US20250197862A1 (en) 2023-12-15 2025-06-19 Genzyme Corporation ARTIFICIAL microRNAs TARGETING TAU
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025160429A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
WO2025160434A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease
WO2025188625A1 (en) 2024-03-03 2025-09-12 Passage Bio, Inc. Recombinant adeno-associated virus for treatment of neurodegenerative disorders
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors
CN118459608B (zh) * 2024-04-26 2025-01-24 泰州博莱得利生物科技有限公司 犬细小病毒特异性免疫制剂、制备方法和应用
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
CN118460614B (zh) * 2024-07-05 2025-03-11 凌意(杭州)生物科技有限公司 一种腺相关病毒Cap蛋白的表达框
CN120249230B (zh) * 2025-06-05 2025-11-07 鼐济医药科技(杭州)有限公司 一种用于同时生产多种重组腺相关病毒的方法

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 (en) 1991-07-10 1993-01-11 Isaac Neuman Article including a container containing at least one precious stone
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
EP0870044B1 (de) * 1995-12-01 2004-04-14 Crucell Holland B.V. Regulierte expression von proteinen in stabil transformierten säugetierzellen
WO1998010087A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
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
WO1998009657A2 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
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
CA2264499A1 (en) * 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
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
WO1998024924A1 (en) * 1996-12-05 1998-06-11 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 (de) * 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methoden und zelllinien für die produktion von rekombinanten adeno-assoziierten viren
AU9774998A (en) 1997-09-19 1999-04-12 Trustees Of The University Of Pennsylvania, The Method for gene transfer using bcl2 and compositions useful therein
CA2303768C (en) * 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
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
ATE286138T1 (de) 1998-03-20 2005-01-15 Univ Pennsylvania Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
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
DK1127150T3 (da) * 1998-11-05 2007-09-24 Univ Pennsylvania Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse
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
AU2409200A (en) 1999-06-02 2000-12-28 Trustees Of The University Of Pennsylvania, The Compositions and methods useful for production of recombinant viruses which require helper viruses
WO2001014539A2 (en) * 1999-08-20 2001-03-01 Johns Hopkins University School Of Medicine Methods and compositions for the construction and use of fusion libraries
EP1218035A2 (de) * 1999-09-29 2002-07-03 The Trustees Of The University Of Pennsylvania Schnelle peg-modifikation von virale vektoren
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
WO2001040455A2 (en) 1999-12-03 2001-06-07 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yields of recombinant adenoviruses using multiple packaging signals
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
WO2001068888A2 (en) * 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
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
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
WO2001083692A2 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
CN1293204C (zh) * 2000-08-30 2007-01-03 戴诺生物技术有限公司 等位基因的测定方法
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
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
JP4810062B2 (ja) * 2001-12-17 2011-11-09 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 アデノ随伴ウイルス（ａａｖ）血清型８の配列
WO2003104392A2 (en) 2001-12-18 2003-12-18 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
US20040002159A1 (en) * 2002-04-05 2004-01-01 Weidong Xiao Methods for the production of chimeric adeno-associated virus (AAV) vectors, compositions of chimeric AAV vectors, and methods of use thereof
EP1359217B1 (de) * 2002-04-29 2006-12-13 The Trustees of The University of Pennsylvania Methode für die direkte Gewinnung und Amplifikation von integrierten Viren aus zellulärer Gewebe-DNA
PL2357189T3 (pl) 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
EP3910063A1 (de) * 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Kladen aus adeno-assoziiertem virus (aav), sequenzen, vektoren damit und verwendungen davon
WO2006039218A2 (en) 2004-09-30 2006-04-13 The Trustees Of The University Of Pennsylvania Perfusion circuit and use therein in targeted delivery of macromolecules
EP3409296A1 (de) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
US7788045B2 (en) 2005-09-01 2010-08-31 Meditasks, Llc Systems and method for homeostatic blood states
JP5268890B2 (ja) 2006-04-28 2013-08-21 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 Ａａｖの規模適応性の製造方法
JP2009102988A (ja) 2007-10-19 2009-05-14 Toyota Motor Corp 内燃機関の燃料噴射装置
AU2009223115B2 (en) 2008-03-14 2015-09-24 Humanzyme, Inc. Recombinant production of authentic human proteins using human cell expression systems
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
EP3540055A1 (de) 2010-04-23 2019-09-18 University of Massachusetts Auf das zns abzielende aav-vektoren und verfahren zur verwendung davon
EP2731470B1 (de) 2011-07-15 2017-09-06 Alfred Kärcher GmbH & Co. KG Rotationsbürste und kehrmaschine mit einer rotationsbürste
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
CN104520428B (zh) 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
US9819463B2 (en) 2016-02-18 2017-11-14 Huawei Technologies Co., Ltd. Method and apparatus for transmitting data in a wireless communication system
US11827906B2 (en) 2017-02-28 2023-11-28 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor

Also Published As

Publication number Publication date
ATE317916T1 (de) 2006-03-15
US11034976B2 (en) 2021-06-15
CA3066428C (en) 2022-05-24
JP2013013414A (ja) 2013-01-24
BR122016004546B8 (pt) 2021-07-27
CA2945734A1 (en) 2003-05-22
SG168422A1 (en) 2011-02-28
CA2756866A1 (en) 2003-05-22
BR0214119A (pt) 2005-06-28
US20210285012A1 (en) 2021-09-16
IL227866A0 (en) 2013-09-30
IL258545B (en) 2019-11-28
CA3066428A1 (en) 2003-05-22
IL233391A (en) 2016-12-29
WO2003042397A3 (en) 2004-02-05
PH12016502583A1 (en) 2017-10-18
US10041090B2 (en) 2018-08-07
BR122016004546B1 (pt) 2016-11-29
CN103555678B (zh) 2018-02-09
IL221602A0 (en) 2012-10-31
JP3958191B2 (ja) 2007-08-15
US11499167B2 (en) 2022-11-15
US20190024117A1 (en) 2019-01-24
ES2258601T3 (es) 2006-09-01
IL234063A (en) 2017-06-29
CN103555676B (zh) 2016-08-31
SG10202108118RA (en) 2021-08-30
NZ600121A (en) 2013-12-20
NO336801B1 (no) 2015-11-02
NO336468B1 (no) 2015-08-31
US20160097040A1 (en) 2016-04-07
PH12014501487B1 (en) 2015-12-07
US20170240921A1 (en) 2017-08-24
US10544432B2 (en) 2020-01-28
MXPA04004600A (es) 2004-08-13
EP1310571A3 (de) 2003-08-13
US11041171B2 (en) 2021-06-22
HK1068925A1 (en) 2005-05-06
NZ532635A (en) 2007-05-31
HU229379B1 (en) 2013-11-28
JP2005525086A (ja) 2005-08-25
CN103555676A (zh) 2014-02-05
US20110263027A1 (en) 2011-10-27
IL270065B1 (en) 2023-06-01
JP2016136953A (ja) 2016-08-04
JP6212142B2 (ja) 2017-10-11
IL213810A0 (en) 2011-07-31
HUP0600229A2 (en) 2006-06-28
US10308958B2 (en) 2019-06-04
CA3159060A1 (en) 2003-05-22
DK1310571T3 (da) 2006-06-19
CN103555678A (zh) 2014-02-05
US20130045186A1 (en) 2013-02-21
MX359371B (es) 2018-09-25
PL397823A1 (pl) 2012-07-16
CN102181480A (zh) 2011-09-14
CA2465868C (en) 2017-02-28
NZ618298A (en) 2015-04-24
JP5669795B2 (ja) 2015-02-18
US20200087684A1 (en) 2020-03-19
CA2864537A1 (en) 2003-05-22
IL213810A (en) 2014-08-31
BR122016004944B1 (pt) 2016-11-22
EP1456419B1 (de) 2011-08-17
IL161827A (en) 2009-11-18
CA2915124A1 (en) 2003-05-22
AU2002361573B2 (en) 2008-06-12
NZ548094A (en) 2008-07-31
US20170298388A1 (en) 2017-10-19
CA2915124C (en) 2018-08-14
ZA200403360B (en) 2005-04-26
KR101014207B1 (ko) 2011-02-14
CN103555677A (zh) 2014-02-05
NO338362B1 (no) 2016-08-15
CA3159060C (en) 2023-05-16
NO334379B1 (no) 2014-02-24
CN103589692B (zh) 2018-07-24
CN105671005A (zh) 2016-06-15
US10508286B2 (en) 2019-12-17
DE60209193D1 (de) 2006-04-20
NZ591012A (en) 2012-06-29
PH12014501487A1 (en) 2015-12-07
IL248724B (en) 2018-04-30
KR20050058234A (ko) 2005-06-16
EP2338900A1 (de) 2011-06-29
BRPI0214119B8 (pt) 2021-05-25
US20220213501A1 (en) 2022-07-07
CA2406745C (en) 2006-01-10
IL270065A (de) 2019-12-31
IL233391A0 (en) 2014-08-31
US20200080109A1 (en) 2020-03-12
SG10201506627PA (en) 2015-10-29
IL258545A (en) 2018-06-28
EP2341068A1 (de) 2011-07-06
NO20042183L (no) 2004-07-14
HU230406B1 (hu) 2016-04-28
CA2406745A1 (en) 2003-05-13
CA2945734C (en) 2020-02-25
NO20150196L (no) 2004-07-14
US20220025401A1 (en) 2022-01-27
SG157224A1 (en) 2009-12-29
US11034977B2 (en) 2021-06-15
MX346493B (es) 2017-03-21
IL161827A0 (en) 2005-11-20
JP2003235562A (ja) 2003-08-26
US9790472B2 (en) 2017-10-17
EP1456419A4 (de) 2005-11-09
EP2341068B1 (de) 2013-09-25
BR122016004944B8 (pt) 2021-07-27
IL221602A (en) 2015-09-24
PL220644B1 (pl) 2015-11-30
CN102181480B (zh) 2016-01-27
EP1456419A2 (de) 2004-09-15
JP5969547B2 (ja) 2016-08-17
CN103589692A (zh) 2014-02-19
PL217623B1 (pl) 2014-08-29
IL227866A (en) 2015-11-30
EP2338900B1 (de) 2014-01-01
KR20100029134A (ko) 2010-03-15
IL248724A0 (en) 2017-01-31
US20030138772A1 (en) 2003-07-24
DE60209193T2 (de) 2006-09-28
JP5140627B2 (ja) 2013-02-06
PL222683B1 (pl) 2016-08-31
PL221877B1 (pl) 2016-06-30
NO20131359L (no) 2004-07-14
NZ578982A (en) 2011-03-31
US8906675B2 (en) 2014-12-09
ES2439515T3 (es) 2014-01-23
US20180030479A1 (en) 2018-02-01
PL373864A1 (en) 2005-09-19
PL404537A1 (pl) 2014-02-17
PL409838A1 (pl) 2015-07-20
IL193525A0 (en) 2009-02-11
US11377669B2 (en) 2022-07-05
NO20140988L (no) 2004-07-14
US8524446B2 (en) 2013-09-03
JP2009195237A (ja) 2009-09-03
WO2003042397A2 (en) 2003-05-22
US10526617B2 (en) 2020-01-07
HUP0600229A3 (en) 2010-01-28
KR101015854B1 (ko) 2011-02-23
JP4677187B2 (ja) 2011-04-27
HK1056198A1 (en) 2004-02-06
EP1310571A2 (de) 2003-05-14
CA2864537C (en) 2016-11-29
IL193525A (en) 2014-08-31
US20110151434A1 (en) 2011-06-23
US20200131534A1 (en) 2020-04-30
SG10201912509RA (en) 2020-02-27
JP2014239680A (ja) 2014-12-25
IL234063A0 (en) 2014-09-30
EP1310571B1 (de) 2006-02-15
IL270065B2 (en) 2023-10-01
NZ564506A (en) 2009-09-25
CA2756866C (en) 2017-06-06
CN105671005B (zh) 2020-09-15
CA2465868A1 (en) 2003-05-22
ES2455126T3 (es) 2014-04-14
CN103555677B (zh) 2018-01-30
BRPI0214119B1 (pt) 2016-09-27

Similar Documents

Publication Publication Date Title
ATE520707T1 (de) Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
ATE469219T1 (de) Zusammensetzungen, methoden und reagentien zur nukleinsäureisolierung mittels oberflächenaktiven substanzen und proteasen
DE69632466D1 (de) Lösung die chaotropische mittel enthält und verwendung dieser lösung in verfahren zur isolierung von dna, rna und proteinen
ATE428003T1 (de) Oligonukleotidprimer zur effizienten detektion von hepatitis c virus (hcv) sowie verwendungsverfahren
BR0111125A (pt) Moléculas de ácido nucléico de nrg-2, polipeptìdeos, e métodos diagnóstico e terapêuticos
ATE345868T1 (de) Zweirichtungs-durchfluss-zentrifugalmikrofluid- vorrichtungen
WO2004072229A3 (en) Chemical treatment of biological samples for nucleic acid extraction and kits therefor
ATE282831T1 (de) Verfahren zur diagnose von sepsis unter bestimmung löslicher cytokeratinfragmente
IL157917A0 (en) Method for sample identification in a mammal as well as a kit for performing this method
WO2005027710A3 (en) Methods for identifying subjects at risk of melanoma and treatments thereof
EP1580265A4 (de) Aus einem neuroblastom in stufe 4s isoliertenukleinsäuren
WO2001007650A3 (en) Improvements in and relating to forensic investigations
WO2003001212A3 (en) A method for diagnosing a person having multiple sclerosis
ATE478147T1 (de) Aus einem neuroblastom isolierte nukleinsäuren
WO2004104170A3 (en) Tools for detecting moraxella catarrhalis.
EP1306446A4 (de) Testsätze zur extraktion von nukleinsäuren und methoden zur extraktion von nukleinsäuren durch verwendung der testsätze
ATA12952001A (de) Verfahren zur genetischen diagnose von hämochromatose sowie sonden zur genetischen diagnose von hämochromatose

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties