AR122404A1 - Variantes de cápside y usos de las mismas - Google Patents

Variantes de cápside y usos de las mismas

Info

Publication number
AR122404A1
AR122404A1 ARP210100790A ARP210100790A AR122404A1 AR 122404 A1 AR122404 A1 AR 122404A1 AR P210100790 A ARP210100790 A AR P210100790A AR P210100790 A ARP210100790 A AR P210100790A AR 122404 A1 AR122404 A1 AR 122404A1
Authority
AR
Argentina
Prior art keywords
aav
variants
transgene
administering
adeno
Prior art date
Application number
ARP210100790A
Other languages
English (en)
Inventor
Guangping Gao
Guangchao Xu
Phillip Tai
Yuquan Wei
Li Luo
Original Assignee
Univ Massachusetts
Univ Sichuan
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 Univ Massachusetts, Univ Sichuan filed Critical Univ Massachusetts
Publication of AR122404A1 publication Critical patent/AR122404A1/es

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (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

Los aspectos de la descripción se relacionan con composiciones y métodos para administrar un transgén (por ejemplo, un transgén que codifica uno o más productos génicos) a una célula objetivo. La descripción se basa, en parte, en variantes de las proteínas de la cápside del virus adenoasociado (AAV) caracterizadas por tropismos para ciertos tipos celulares (por ejemplo, neuronas, células de la musculatura, células óseas, células cardíacas, etc.). En algunas realizaciones, los AAVs recombinantes (rAAVs) que comprenden las variantes de proteínas de la cápside (por ejemplo, AAVv66, SEC ID Nº 1) son empaquetadas más eficazmente que los rAAVs que tienen ciertas proteínas de la cápside del AAV del tipo salvaje. La descripción describe además métodos para administrar un rAAV que comprende las variantes de las proteínas de la cápside de AAV. Reivindicación 1: Un método para administrar un transgén a una célula objetivo en un sujeto, caracterizado porque comprende administrar intracranealmente al sujeto un virus adenoasociado recombinante (rAAV) que comprende: (i) un ácido nucleico aislado que comprende un transgén que codifica uno o más productos génicos de interés; y (ii) una proteína de la cápside de ácido adenoasociado (AAV) que tiene la secuencia expuesta en SEC ID Nº 1.
ARP210100790A 2020-03-31 2021-03-30 Variantes de cápside y usos de las mismas AR122404A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063003143P 2020-03-31 2020-03-31

Publications (1)

Publication Number Publication Date
AR122404A1 true AR122404A1 (es) 2022-09-07

Family

ID=75870702

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210100790A AR122404A1 (es) 2020-03-31 2021-03-30 Variantes de cápside y usos de las mismas

Country Status (14)

Country Link
US (1) US20230138766A1 (es)
EP (1) EP4126911A1 (es)
JP (1) JP2023529054A (es)
KR (1) KR20230113689A (es)
CN (1) CN115698039A (es)
AR (1) AR122404A1 (es)
AU (1) AU2021248577A1 (es)
BR (1) BR112022019304A2 (es)
CA (1) CA3177182A1 (es)
CO (1) CO2022015313A2 (es)
IL (1) IL296765A (es)
MX (1) MX2022012279A (es)
TW (1) TW202204377A (es)
WO (1) WO2021202494A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2023060264A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2024020376A1 (en) * 2022-07-22 2024-01-25 University Of Massachusetts Aav-mediated delivery of osteoblast/osteoclast-regulating mirnas for osteoporosis therapy

Family Cites Families (25)

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US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
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EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
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US20200316221A1 (en) * 2016-10-13 2020-10-08 University Of Massachusetts Aav capsid designs
KR20190075964A (ko) * 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계

Also Published As

Publication number Publication date
IL296765A (en) 2022-11-01
AU2021248577A1 (en) 2022-09-29
EP4126911A1 (en) 2023-02-08
CA3177182A1 (en) 2021-10-07
TW202204377A (zh) 2022-02-01
CO2022015313A2 (es) 2023-02-16
CN115698039A (zh) 2023-02-03
BR112022019304A2 (pt) 2022-12-06
MX2022012279A (es) 2023-02-23
JP2023529054A (ja) 2023-07-07
KR20230113689A (ko) 2023-08-01
US20230138766A1 (en) 2023-05-04
WO2021202494A1 (en) 2021-10-07

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