BR112021019436A2 - Vetores de vírus adeno-associados (aav) modificados para expressão transgênica - Google Patents

Vetores de vírus adeno-associados (aav) modificados para expressão transgênica

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Publication number
BR112021019436A2
BR112021019436A2 BR112021019436A BR112021019436A BR112021019436A2 BR 112021019436 A2 BR112021019436 A2 BR 112021019436A2 BR 112021019436 A BR112021019436 A BR 112021019436A BR 112021019436 A BR112021019436 A BR 112021019436A BR 112021019436 A2 BR112021019436 A2 BR 112021019436A2
Authority
BR
Brazil
Prior art keywords
vectors
aav
transgene expression
associated virus
modified
Prior art date
Application number
BR112021019436A
Other languages
English (en)
Inventor
A Maguire Casey
Marie Hudry Eloise
S Hanlon Killian
Original Assignee
Harvard College
Massachusetts Gen Hospital
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Harvard College, Massachusetts Gen Hospital filed Critical Harvard College
Publication of BR112021019436A2 publication Critical patent/BR112021019436A2/pt

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—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
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1037—Screening libraries presented on the surface of microorganisms, e.g. phage display, E. coli display
    • 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
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/02—Libraries contained in or displayed by microorganisms, e.g. bacteria or animal cells; Libraries contained in or displayed by vectors, e.g. plasmids; Libraries containing only microorganisms or vectors

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

vetores de vírus adeno-associados (aav) modificados para expressão transgênica. vetores de aav modificados para expressão transgênica, por exemplo, no snc, snp, ouvido interno, coração ou retina, e métodos de uso dos mesmos. também são providos métodos para descobrir novos vetores de aav modificados que medeiam a expressão transgênica em tipos de células desejados.
BR112021019436A 2019-03-28 2020-03-30 Vetores de vírus adeno-associados (aav) modificados para expressão transgênica BR112021019436A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962825703P 2019-03-28 2019-03-28
PCT/US2020/025720 WO2020198737A1 (en) 2019-03-28 2020-03-30 Engineered adeno-associated (aav) vectors for transgene expression

Publications (1)

Publication Number Publication Date
BR112021019436A2 true BR112021019436A2 (pt) 2021-12-07

Family

ID=72609527

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021019436A BR112021019436A2 (pt) 2019-03-28 2020-03-30 Vetores de vírus adeno-associados (aav) modificados para expressão transgênica

Country Status (12)

Country Link
US (1) US20220195458A1 (pt)
EP (1) EP3947422A4 (pt)
JP (1) JP2022527917A (pt)
KR (1) KR20210143869A (pt)
CN (1) CN113874387A (pt)
AU (1) AU2020248116A1 (pt)
BR (1) BR112021019436A2 (pt)
CA (1) CA3135292A1 (pt)
IL (1) IL286725A (pt)
MX (1) MX2021011701A (pt)
SG (1) SG11202110165XA (pt)
WO (1) WO2020198737A1 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019226516A1 (en) 2018-05-24 2019-11-28 Celanese EVA Performance Polymers Corporation Implantable device for sustained release of a macromolecular drug compound
CN111989068A (zh) 2018-05-24 2020-11-24 塞拉尼斯伊娃高性能聚合物公司 用于持续释放大分子药物化合物的可植入器件
AU2019346447A1 (en) 2018-09-26 2021-04-29 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
CA3171166A1 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
IL309742A (en) 2021-07-08 2024-02-01 Tenaya Therapeutics Inc Optimal expression cassettes for gene therapy
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060021087A1 (en) * 2004-04-09 2006-01-26 Baum James A Compositions and methods for control of insect infestations in plants
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
SG10201809075XA (en) * 2013-07-22 2018-11-29 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
ES2739288T3 (es) * 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
CA3234028A1 (en) * 2014-03-11 2015-09-17 Wayne State University A modified mglur6 promoter and methods of use
GB2545763A (en) * 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
US11826433B2 (en) * 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
EP3491008A2 (en) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
EP3662060A2 (en) * 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN110709511A (zh) * 2017-08-28 2020-01-17 加利福尼亚大学董事会 腺相关病毒衣壳变体及其使用方法
CN109207520B (zh) * 2018-09-25 2022-05-10 北京锦篮基因科技有限公司 Leber遗传性视神经病变的基因药物

Also Published As

Publication number Publication date
JP2022527917A (ja) 2022-06-07
AU2020248116A1 (en) 2021-10-14
CA3135292A1 (en) 2020-10-01
US20220195458A1 (en) 2022-06-23
KR20210143869A (ko) 2021-11-29
IL286725A (en) 2021-12-01
CN113874387A (zh) 2021-12-31
EP3947422A4 (en) 2023-05-17
EP3947422A1 (en) 2022-02-09
MX2021011701A (es) 2021-12-10
WO2020198737A1 (en) 2020-10-01
SG11202110165XA (en) 2021-10-28

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