MX2022012279A - Variantes de capsides de aav y usos de los mismos. - Google Patents

Variantes de capsides de aav y usos de los mismos.

Info

Publication number
MX2022012279A
MX2022012279A MX2022012279A MX2022012279A MX2022012279A MX 2022012279 A MX2022012279 A MX 2022012279A MX 2022012279 A MX2022012279 A MX 2022012279A MX 2022012279 A MX2022012279 A MX 2022012279A MX 2022012279 A MX2022012279 A MX 2022012279A
Authority
MX
Mexico
Prior art keywords
protein variants
capsid protein
disclosure
aav
variants
Prior art date
Application number
MX2022012279A
Other languages
English (en)
Inventor
Li Luo
Guangping Gao
Guangchao Xu
Phillip Tai
Yuquan Wei
Original Assignee
Univ Massachusetts
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 filed Critical Univ Massachusetts
Publication of MX2022012279A publication Critical patent/MX2022012279A/es

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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
    • 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
    • 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 Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Aspectos de la divulgación se refieren a composiciones y métodos para entrega de un transgén (por ejemplo, un transgén que codifica uno o más productos génicos) a una célula blanco. La divulgación se basa, en parte, en variantes de la proteína de la cápside del virus adenoasociado (AAV) caracterizadas por tropismos para ciertos tipos de células (por ejemplo, neuronas, células musculares, células óseas, células cardíacas, etc.). En algunas modalidades, los AAV recombinantes (rAAV) que comprenden las variantes de la proteína de la cápside (por ejemplo, AAVv66, SEQ ID NO: 1) se empacan de manera más eficiente que los rAAV que tienen ciertas proteínas de la cápside de AAV tipo silvestre. Los métodos para entrega de un rAAV que comprende las variantes de proteína de la cápside de AAV también se describen en la divulgación.
MX2022012279A 2020-03-31 2021-03-30 Variantes de capsides de aav y usos de los mismos. MX2022012279A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063003143P 2020-03-31 2020-03-31
PCT/US2021/024837 WO2021202494A1 (en) 2020-03-31 2021-03-30 Aav capsids variants and uses thereof

Publications (1)

Publication Number Publication Date
MX2022012279A true MX2022012279A (es) 2023-02-23

Family

ID=75870702

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022012279A MX2022012279A (es) 2020-03-31 2021-03-30 Variantes de capsides de aav y usos de los mismos.

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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP3768386A4 (en) 2018-03-23 2022-04-13 University of Massachusetts GENE THERAPY FOR THE TREATMENT OF BONE DISORDERS
EP4413147A4 (en) 2021-10-08 2025-07-30 Dyno Therapeutics Inc CAPSID VARIANTS AND METHODS OF USE THEREOF
WO2024020376A1 (en) * 2022-07-22 2024-01-25 University Of Massachusetts Aav-mediated delivery of osteoblast/osteoclast-regulating mirnas for osteoporosis therapy

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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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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
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CA3040483A1 (en) * 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
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Also Published As

Publication number Publication date
EP4126911A1 (en) 2023-02-08
CO2022015313A2 (es) 2023-02-16
IL296765A (en) 2022-11-01
WO2021202494A1 (en) 2021-10-07
AU2021248577A1 (en) 2022-09-29
CN115698039A (zh) 2023-02-03
CA3177182A1 (en) 2021-10-07
KR20230113689A (ko) 2023-08-01
TW202204377A (zh) 2022-02-01
US20230138766A1 (en) 2023-05-04
JP2023529054A (ja) 2023-07-07
BR112022019304A2 (pt) 2022-12-06
AR122404A1 (es) 2022-09-07

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