CN116171325A - 用于eEF1A2的基因疗法载体及其用途 - Google Patents

用于eEF1A2的基因疗法载体及其用途 Download PDF

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Publication number
CN116171325A
CN116171325A CN202180056421.4A CN202180056421A CN116171325A CN 116171325 A CN116171325 A CN 116171325A CN 202180056421 A CN202180056421 A CN 202180056421A CN 116171325 A CN116171325 A CN 116171325A
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eef1a2
polynucleotide sequence
raav
seq
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Chinese (zh)
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S·N·沃丁顿
R·卡达
C·D·赫尔佐格
J·恩格
C·萨克拉门多
S·肖尔格
D·里克斯
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UCL Business Ltd
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UCL Business Ltd
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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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    • C12N2830/00—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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  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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CN202180056421.4A 2020-07-23 2021-07-21 用于eEF1A2的基因疗法载体及其用途 Pending CN116171325A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063055775P 2020-07-23 2020-07-23
US63/055,775 2020-07-23
PCT/EP2021/070455 WO2022018171A1 (en) 2020-07-23 2021-07-21 GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF

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CN116171325A true CN116171325A (zh) 2023-05-26

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US (1) US20230330265A1 (https=)
EP (1) EP4185332A1 (https=)
JP (1) JP2023535576A (https=)
KR (1) KR20230039669A (https=)
CN (1) CN116171325A (https=)
AU (1) AU2021312320A1 (https=)
CA (1) CA3186700A1 (https=)
IL (1) IL299955A (https=)
MX (1) MX2023000995A (https=)
WO (2) WO2022017630A1 (https=)

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HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
WO2026006785A2 (en) * 2024-06-28 2026-01-02 Adverum Biotechnologies, Inc. Melanopsin variants for vision restoration
WO2026044087A1 (en) 2024-08-21 2026-02-26 Shape Therapeutics Inc. Increased cellular stability for aav production
WO2026073032A1 (en) 2024-09-27 2026-04-02 Shape Therapeutics Inc. Constructs for improved aav production

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ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
US7122350B2 (en) * 2003-12-12 2006-10-17 Zymogenetics, Inc. Methods for enhancing expression of recombinant proteins
EP2176283B1 (en) 2007-07-14 2016-11-02 University of Iowa Research Foundation Methods and compositions for treating brain diseases
JP2013523776A (ja) 2010-04-02 2013-06-17 セレジーン インコーポレイテッド パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
EP3285788B1 (en) * 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
AU2016324317A1 (en) * 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
KR102918544B1 (ko) * 2018-07-12 2026-01-27 로켓 파마슈티컬스, 리미티드 다논병을 치료하기 위한 유전자 요법 벡터
CA3120923A1 (en) * 2018-11-26 2020-06-04 Universitat Autonoma De Barcelona Fibroblast growth factor 21 (fgf21) gene therapy

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WO2022018171A1 (en) 2022-01-27
US20230330265A1 (en) 2023-10-19
WO2022017630A1 (en) 2022-01-27
AU2021312320A1 (en) 2023-03-16
IL299955A (en) 2023-03-01
EP4185332A1 (en) 2023-05-31
JP2023535576A (ja) 2023-08-18
MX2023000995A (es) 2023-06-16
KR20230039669A (ko) 2023-03-21
CA3186700A1 (en) 2022-01-27

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