CN116568338A - 改进的aav介导的x连锁视网膜劈裂症治疗 - Google Patents

改进的aav介导的x连锁视网膜劈裂症治疗 Download PDF

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Publication number
CN116568338A
CN116568338A CN202180066643.4A CN202180066643A CN116568338A CN 116568338 A CN116568338 A CN 116568338A CN 202180066643 A CN202180066643 A CN 202180066643A CN 116568338 A CN116568338 A CN 116568338A
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vector
sequence
raav
nucleic acid
seq
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CN202180066643.4A
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Chinese (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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University of Florida Research Foundation Inc
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Transition And Organic Metals Composition Catalysts For Addition Polymerization (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202180066643.4A 2020-07-29 2021-07-28 改进的aav介导的x连锁视网膜劈裂症治疗 Pending CN116568338A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063058437P 2020-07-29 2020-07-29
US63/058,437 2020-07-29
PCT/US2021/043582 WO2022026632A2 (en) 2020-07-29 2021-07-28 Improved aav-mediated x-linked retinoschisis therapies

Publications (1)

Publication Number Publication Date
CN116568338A true CN116568338A (zh) 2023-08-08

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US (1) US20230265455A1 (https=)
EP (1) EP4188450A2 (https=)
JP (1) JP2023535956A (https=)
CN (1) CN116568338A (https=)
AU (1) AU2021319065A1 (https=)
CA (1) CA3190214A1 (https=)
IL (1) IL299927A (https=)
WO (1) WO2022026632A2 (https=)

Families Citing this family (9)

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Publication number Priority date Publication date Assignee Title
US20240124893A1 (en) * 2021-11-03 2024-04-18 Teamedon International, Inc. Methods of Treating Human X-Linked Retinoschisis Using Gene Therapy
EP4499847A4 (en) * 2022-03-30 2026-03-25 Univ Pittsburgh Commonwealth Sys Higher Education Adeno-associated viral vectors for the delivery of nucleic acids across retinal regions
CN114848850B (zh) * 2022-04-28 2023-12-15 武汉中眸生物科技有限责任公司 Rs1基因在制备xlrs治疗剂中的应用及治疗剂
CN118359687A (zh) * 2023-01-18 2024-07-19 上海朗昇生物科技有限公司 一种新衣壳蛋白变体以及其用途
EP4704920A1 (en) * 2023-05-04 2026-03-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vectors for multipartite gene delivery and stargardt disease treatment
WO2025022144A1 (en) * 2023-07-26 2025-01-30 Sorbonne Universite Chimeric promoter for targeted expression in aii amacrine cells
WO2025091025A1 (en) * 2023-10-26 2025-05-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders relating to sirt1
WO2025113432A1 (zh) * 2023-11-27 2025-06-05 北京因诺惟康医药科技有限公司 一种用于治疗x染色体连锁视网膜劈裂症的基因药物
WO2025231339A1 (en) * 2024-05-02 2025-11-06 Avista Therapeutics, Inc. Adeno-associated virus vectors for therapeutic nucleic acid delivery to retinal cells

Family Cites Families (16)

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AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9567376B2 (en) * 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
WO2014127196A1 (en) * 2013-02-15 2014-08-21 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Aav8 retinoschisin expression vector for treating x-linked retinoschisis
HK1220488A1 (zh) * 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
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
EP3800191B1 (en) * 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
PL3628334T3 (pl) * 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej
US10533187B2 (en) 2015-03-18 2020-01-14 University Of Florida Research Foundation, Incorporated Methods and compositions for restoration of cone function in BCM
CA3001594A1 (en) * 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
WO2017070491A1 (en) * 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
US20210228738A1 (en) * 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
US20200390907A1 (en) * 2018-01-22 2020-12-17 The Usa, As Represented By The Secretary, Department Of Health And Human Services Methods and compositions for treating genetically linked diseases of the eye
US12605466B2 (en) * 2019-03-04 2026-04-21 University Of Florida Research Foundation, Incorporated Enhanced human opsin promoter for rod specific expression
US20220403417A1 (en) * 2019-11-20 2022-12-22 University Of Massachusetts Aav-based delivery of thymine kinase 2

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Publication number Publication date
EP4188450A2 (en) 2023-06-07
WO2022026632A3 (en) 2022-03-10
AU2021319065A1 (en) 2023-02-16
CA3190214A1 (en) 2022-02-03
JP2023535956A (ja) 2023-08-22
US20230265455A1 (en) 2023-08-24
WO2022026632A2 (en) 2022-02-03
IL299927A (en) 2023-03-01

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