CN113347962B - 通过合理设计增强的新型aav病毒在视网膜中的高效转导和侧向扩散 - Google Patents

通过合理设计增强的新型aav病毒在视网膜中的高效转导和侧向扩散 Download PDF

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CN113347962B
CN113347962B CN202080010452.1A CN202080010452A CN113347962B CN 113347962 B CN113347962 B CN 113347962B CN 202080010452 A CN202080010452 A CN 202080010452A CN 113347962 B CN113347962 B CN 113347962B
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CN113347962A (zh
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香农·E·博耶
桑福德·L·博耶
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University of Florida Research Foundation Inc
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    • 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
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    • 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
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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

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  • Gastroenterology & Hepatology (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202080010452.1A 2019-01-23 2020-01-23 通过合理设计增强的新型aav病毒在视网膜中的高效转导和侧向扩散 Active CN113347962B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962795695P 2019-01-23 2019-01-23
US62/795,695 2019-01-23
PCT/US2020/014838 WO2020154535A1 (en) 2019-01-23 2020-01-23 Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design

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CN113347962A CN113347962A (zh) 2021-09-03
CN113347962B true CN113347962B (zh) 2025-01-28

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US (1) US20220133909A1 (enExample)
EP (1) EP3914229A4 (enExample)
JP (1) JP7624727B2 (enExample)
CN (1) CN113347962B (enExample)
AU (1) AU2020212026B2 (enExample)
CA (1) CA3125294A1 (enExample)
WO (1) WO2020154535A1 (enExample)

Families Citing this family (1)

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WO2024223696A1 (en) * 2023-04-24 2024-10-31 Universiteit Gent Compounds to treat inherited retinal disease

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106661591A (zh) * 2014-05-02 2017-05-10 建新公司 用于视网膜及cns基因疗法的aav载体
CN106906241A (zh) * 2005-04-07 2017-06-30 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU7467598A (en) * 1997-04-21 1998-11-13 University Of Florida Materials and methods for treatment of retinal diseases
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
AU2011242527B2 (en) * 2010-04-23 2016-05-19 University Of Florida Research Foundation, Inc. rAAV-guanylate cyclase compositions and methods for treating Leber's congenital amaurosis-1 (LCA1)
EP3113787B1 (en) * 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
US10533187B2 (en) 2015-03-18 2020-01-14 University Of Florida Research Foundation, Incorporated Methods and compositions for restoration of cone function in BCM
CA2985786A1 (en) 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
US20170348387A1 (en) * 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy
EP3491008A2 (en) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
WO2018156654A1 (en) 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106906241A (zh) * 2005-04-07 2017-06-30 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
CN106661591A (zh) * 2014-05-02 2017-05-10 建新公司 用于视网膜及cns基因疗法的aav载体

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Publication number Publication date
JP7624727B2 (ja) 2025-01-31
CN113347962A (zh) 2021-09-03
US20220133909A1 (en) 2022-05-05
WO2020154535A1 (en) 2020-07-30
EP3914229A4 (en) 2022-11-02
AU2020212026B2 (en) 2025-10-09
CA3125294A1 (en) 2020-07-30
JP2022523050A (ja) 2022-04-21
EP3914229A1 (en) 2021-12-01
AU2020212026A1 (en) 2021-07-22

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