MX2021011701A - Vectores adenoasociados fabricados por ingenieria (aav) para expresion transgenica. - Google Patents

Vectores adenoasociados fabricados por ingenieria (aav) para expresion transgenica.

Info

Publication number
MX2021011701A
MX2021011701A MX2021011701A MX2021011701A MX2021011701A MX 2021011701 A MX2021011701 A MX 2021011701A MX 2021011701 A MX2021011701 A MX 2021011701A MX 2021011701 A MX2021011701 A MX 2021011701A MX 2021011701 A MX2021011701 A MX 2021011701A
Authority
MX
Mexico
Prior art keywords
transgene expression
vectors
aav
engineered adeno
engineered
Prior art date
Application number
MX2021011701A
Other languages
English (en)
Inventor
Casey A Maguire
Eloise Marie Hudry
Killian S Hanlon
Original Assignee
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 Massachusetts Gen Hospital filed Critical Massachusetts Gen Hospital
Publication of MX2021011701A publication Critical patent/MX2021011701A/es

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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
    • 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
    • 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
    • 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
    • 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

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)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Gastroenterology & Hepatology (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

Se describen vectores AAV fabricados por ingeniería para su expresión transgénica, por ejemplo, en el SNC, SNP, oído interno, corazón, o retina, y métodos de uso de los mismos. También se proporcionan métodos para descubrir nuevos vectores AAV fabricados por ingeniería que median la expresión transgénica en tipos de células deseados.
MX2021011701A 2019-03-28 2020-03-30 Vectores adenoasociados fabricados por ingenieria (aav) para expresion transgenica. MX2021011701A (es)

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
MX2021011701A true MX2021011701A (es) 2021-12-10

Family

ID=72609527

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021011701A MX2021011701A (es) 2019-03-28 2020-03-30 Vectores adenoasociados fabricados por ingenieria (aav) para expresion transgenica.

Country Status (12)

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
JP2021524841A (ja) 2018-05-24 2021-09-16 セラニーズ・イーブイエイ・パフォーマンス・ポリマーズ・エルエルシー 高分子薬剤化合物の持続放出用の埋め込み型デバイス
MX2020012459A (es) 2018-05-24 2021-04-28 Celanese Eva Performance Polymers Llc Dispositivo implantable para liberacion sostenida de un compuesto de farmaco macromolecular.
CA3114199A1 (en) 2018-09-26 2020-04-02 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
JP2023513749A (ja) 2020-02-13 2023-04-03 テナヤ セラピューティクス， インコーポレイテッド 心疾患を治療するための遺伝子療法ベクター
AR126407A1 (es) 2021-07-08 2023-10-11 Tenaya Therapeutics Inc Casetes de expresión optimizada para genoterapia
TW202421787A (zh) 2022-09-06 2024-06-01 美商特納亞治療股份有限公司 保護心臟之心臟病療法

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR048685A1 (es) * 2004-04-09 2006-05-17 Monsanto Technology Llc Metodos para el control de infestaciones de insectos en plantas.
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
HUE047996T2 (hu) * 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
ES2739288T3 (es) * 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
US10307492B2 (en) * 2014-03-11 2019-06-04 Wayne State University Modified MGLUR6 promoter and methods of use
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
GB2545763A (en) * 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
CA3011939A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
SG11201811603WA (en) * 2016-07-26 2019-02-27 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
KR20200044793A (ko) * 2017-08-03 2020-04-29 보이저 테라퓨틱스, 인크. Aav의 전달을 위한 조성물 및 방법
JP2020534788A (ja) * 2017-08-28 2020-12-03 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 アデノ随伴ウイルスカプシド変異体及びその使用方法
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
CA3135292A1 (en) 2020-10-01
SG11202110165XA (en) 2021-10-28
WO2020198737A1 (en) 2020-10-01
EP3947422A1 (en) 2022-02-09
IL286725A (en) 2021-12-01
KR20210143869A (ko) 2021-11-29
US20220195458A1 (en) 2022-06-23
BR112021019436A2 (pt) 2021-12-07
EP3947422A4 (en) 2023-05-17
AU2020248116A1 (en) 2021-10-14
CN113874387A (zh) 2021-12-31

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