CN115896106A - 用于治疗显性视网膜色素变性的aav载体 - Google Patents

用于治疗显性视网膜色素变性的aav载体 Download PDF

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Publication number
CN115896106A
CN115896106A CN202210934990.0A CN202210934990A CN115896106A CN 115896106 A CN115896106 A CN 115896106A CN 202210934990 A CN202210934990 A CN 202210934990A CN 115896106 A CN115896106 A CN 115896106A
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rna
rho
sequence
seq
vector
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阿尔弗雷德·S·莱温
威廉·W·豪斯维特
迈克尔·T·马森吉尔
威廉·贝尔特兰
古斯塔沃·D·阿吉雷
阿图尔·西代西扬
塞缪尔·雅各布森
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University of Pennsylvania Penn
University of Florida Research Foundation Inc
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University of Pennsylvania Penn
University of Florida Research Foundation Inc
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    • 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
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CN202210934990.0A 2016-03-01 2017-03-01 用于治疗显性视网膜色素变性的aav载体 Pending CN115896106A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201662302122P 2016-03-01 2016-03-01
US62/302,122 2016-03-01
US201662398451P 2016-09-22 2016-09-22
US62/398,451 2016-09-22
PCT/US2017/020289 WO2017151823A1 (en) 2016-03-01 2017-03-01 Aav vectors for treatment of dominant retinitis pigmentosa
CN201780014309.8A CN109154002B (zh) 2016-03-01 2017-03-01 用于治疗显性视网膜色素变性的aav载体

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US (2) US11118185B2 (https=)
EP (2) EP3423582B1 (https=)
JP (3) JP7272795B2 (https=)
CN (2) CN115896106A (https=)
AU (2) AU2017227776C1 (https=)
CA (1) CA3014671A1 (https=)
ES (1) ES2923877T3 (https=)
IL (2) IL302455A (https=)
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CN115896106A (zh) 2016-03-01 2023-04-04 佛罗里达大学研究基金会有限公司 用于治疗显性视网膜色素变性的aav载体
JP7385282B2 (ja) * 2017-11-07 2023-11-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 環状ｒｎａ分子のための方法及び組成物
BR112020023298A2 (pt) * 2018-05-15 2021-03-09 University Of Washington composições e métodos para reduzir a spliceopatia e tratamento de distúrbios de dominância de rna
CN112204145B (zh) * 2018-06-01 2024-03-26 佛罗里达大学研究基金会有限公司 用于治疗显性视网膜色素变性的组合物和方法
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
EP4013414A4 (en) * 2019-08-15 2023-09-27 The Children's Hospital of Philadelphia COMBINED INTRON-DERIVED MIRNA AND TRANSGENE THERAPY FOR THE TREATMENT OF SCA1
US20230149439A1 (en) * 2020-04-21 2023-05-18 Chigenovo Co., Ltd. Rho-adrp gene editing-based methods and compositions
CN111926044B (zh) * 2020-10-12 2021-01-22 北京大学第三医院（北京大学第三临床医学院） 结合突变rho基因的核酸分子和试剂盒
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2025231487A1 (en) * 2024-05-03 2025-11-06 The Trustees Of The University Of Pennsylvania Photoreceptor specific promotors for use in gene therapies for treatment of retinal degeneration

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CN102918152A (zh) * 2010-04-23 2013-02-06 佛罗里达大学研究基金公司 用于治疗莱伯氏先天性黑蒙-1（lca1）的raav-鸟苷酸环化酶组合物及方法
US20150030572A1 (en) * 2012-03-09 2015-01-29 The Johns Hopkins University Identification of molecular pathways and methods of use thereof for treating retinal neurodegeneration and other neurodegenerative disorders
CN104470545A (zh) * 2012-05-04 2015-03-25 诺华股份有限公司 用于治疗视网膜营养不良的病毒载体

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US20170348387A1 (en) 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy
CN115896106A (zh) 2016-03-01 2023-04-04 佛罗里达大学研究基金会有限公司 用于治疗显性视网膜色素变性的aav载体

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WO2005079815A2 (en) * 2004-02-18 2005-09-01 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin Methods and reagents for treating autosomal dominant diseases of the eye
CN102918152A (zh) * 2010-04-23 2013-02-06 佛罗里达大学研究基金公司 用于治疗莱伯氏先天性黑蒙-1（lca1）的raav-鸟苷酸环化酶组合物及方法
US20150030572A1 (en) * 2012-03-09 2015-01-29 The Johns Hopkins University Identification of molecular pathways and methods of use thereof for treating retinal neurodegeneration and other neurodegenerative disorders
CN104470545A (zh) * 2012-05-04 2015-03-25 诺华股份有限公司 用于治疗视网膜营养不良的病毒载体

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AU2023237106B2 (en) 2025-08-28
JP2019511216A (ja) 2019-04-25
EP3423582A4 (en) 2019-11-13
US11118185B2 (en) 2021-09-14
EP3423582A1 (en) 2019-01-09
EP3423582B1 (en) 2022-05-04
CN109154002A (zh) 2019-01-04
JP2025016451A (ja) 2025-02-04
IL261014A (en) 2018-10-31
JP7642211B2 (ja) 2025-03-10
JP2022185052A (ja) 2022-12-13
JP7272795B2 (ja) 2023-05-12
AU2023237106A1 (en) 2023-10-19
WO2017151823A1 (en) 2017-09-08
EP4119668A1 (en) 2023-01-18
IL261014B1 (en) 2023-06-01
IL302455A (en) 2023-06-01
US20190093111A1 (en) 2019-03-28
ES2923877T3 (es) 2022-10-03
CN109154002B (zh) 2022-08-30
AU2017227776C1 (en) 2024-01-25
US20210324387A1 (en) 2021-10-21
CA3014671A1 (en) 2017-09-08
AU2017227776B2 (en) 2023-07-06
IL261014B2 (en) 2023-10-01

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