CN113423434A - 用于基因递送的重组腺相关病毒载体 - Google Patents

用于基因递送的重组腺相关病毒载体 Download PDF

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Publication number
CN113423434A
CN113423434A CN201980088032.2A CN201980088032A CN113423434A CN 113423434 A CN113423434 A CN 113423434A CN 201980088032 A CN201980088032 A CN 201980088032A CN 113423434 A CN113423434 A CN 113423434A
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aav
seq
promoter
leu
gly
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T.J.米勒
L.帕德吉马斯
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Abe Onna Therapeutics Ltd
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CN201980088032.2A 2018-12-05 2019-12-04 用于基因递送的重组腺相关病毒载体 Pending CN113423434A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862775871P 2018-12-05 2018-12-05
US62/775,871 2018-12-05
US201962801195P 2019-02-05 2019-02-05
US62/801,195 2019-02-05
US201962863126P 2019-06-18 2019-06-18
US62/863,126 2019-06-18
US201962914856P 2019-10-14 2019-10-14
US62/914,856 2019-10-14
PCT/US2019/064396 WO2020117898A1 (en) 2018-12-05 2019-12-04 Recombinant adeno-associated viral vector for gene delivery

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CN113423434A true CN113423434A (zh) 2021-09-21

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Country Link
US (1) US12454701B2 (https=)
EP (1) EP3890786A4 (https=)
JP (2) JP7590968B2 (https=)
KR (1) KR20220022107A (https=)
CN (1) CN113423434A (https=)
AU (1) AU2019391042A1 (https=)
BR (1) BR112021009913A2 (https=)
CA (1) CA3121177A1 (https=)
IL (1) IL283546A (https=)
MX (1) MX2021006646A (https=)
WO (1) WO2020117898A1 (https=)

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CN113025618A (zh) * 2019-12-24 2021-06-25 上海市第一人民医院 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用
CN114181318A (zh) * 2021-11-08 2022-03-15 四川大学 一种高效组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用
CN115029360A (zh) * 2022-05-30 2022-09-09 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒
CN116622750A (zh) * 2023-06-01 2023-08-22 上海勉亦生物科技有限公司 优化的人法布里转基因表达盒及其用途
WO2023202637A1 (en) * 2022-04-19 2023-10-26 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating neurodegenerative disorders
WO2024077815A1 (zh) * 2022-10-09 2024-04-18 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024217422A1 (en) * 2023-04-17 2024-10-24 Exegenesis Bio Co. Compositions and methods for gene therapies
WO2024235159A1 (zh) * 2023-05-12 2024-11-21 上海金珂博生物技术有限公司 一种携带smn基因表达框的病毒载体及其用途
WO2025039622A1 (zh) * 2023-08-23 2025-02-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

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KR20230117327A (ko) * 2020-09-03 2023-08-08 어빈 에스.와이. 첸 가용성 알칼리성 포스파타제 작제물 및 가용성 알칼리성 포스파타제 작제물을 인코딩하는 폴리뉴클레오티드를 포함하는 발현 벡터
CN114507692B (zh) * 2020-11-16 2024-07-12 舒泰神（北京）生物制药股份有限公司 用于治疗法布里病的腺相关病毒载体及其用途
EP4244360A4 (en) * 2020-11-16 2025-05-07 Beijing SoloBio Genetechnology Company Ltd. Recombinant adeno-associated viruses with enhanced liver tropism and uses thereof
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JP2024519819A (ja) * 2021-05-18 2024-05-21 アビオナ・セラピュ―ティクス・インコーポレイテッド 眼疾患および障害を処置するための方法および組成物
KR20240032971A (ko) 2021-07-08 2024-03-12 테나야 테라퓨틱스, 인코포레이티드 유전자 요법을 위한 최적화된 발현 카세트
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AR126877A1 (es) * 2021-08-25 2023-11-22 Canbridge Pharmaceuticals Inc PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GLUCOSIDASA ÁCIDA (GAA) Y SU USO PARA TRATAR LA ENFERMEDAD DE POMPE
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KR20240133693A (ko) * 2021-11-12 2024-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 점액다당류증 iiia의 치료를 위한 유전자 요법
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KR20240086741A (ko) * 2022-11-28 2024-06-19 (주)지뉴인텍 Pde6b 유전자가 포함된 아데노연관바이러스 재조합 벡터를 유효성분으로 포함하는 유전성 망막질환 예방 또는 치료용 조성물
IT202300004443A1 (it) * 2023-03-09 2024-09-09 Int Centre For Genetic Engineering And Biotechnology Icgeb Sequenza codificante per alfa galattosidasi a umana per il trattamento della malattia di fabry
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
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Citations (2)

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