JP7272795B2 - 優性網膜色素変性の治療のためのａａｖベクター - Google Patents

優性網膜色素変性の治療のためのａａｖベクター Download PDF

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JP7272795B2
JP7272795B2 JP2018545818A JP2018545818A JP7272795B2 JP 7272795 B2 JP7272795 B2 JP 7272795B2 JP 2018545818 A JP2018545818 A JP 2018545818A JP 2018545818 A JP2018545818 A JP 2018545818A JP 7272795 B2 JP7272795 B2 JP 7272795B2
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JP2019511216A5 (enExample
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アルフレッド・エス・ルウィン
ウィリアム・ダブリュー・ハウスワース
マイケル・ティ・マッセンギル
ウィリアム・ベルトラン
グスタボ・ディ・アギーレ
アルトゥール・シデシヤン
サミュエル・ジェイコブソン
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ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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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JP2018545818A 2016-03-01 2017-03-01 優性網膜色素変性の治療のためのａａｖベクター Active JP7272795B2 (ja)

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JP2022156496A JP7642211B2 (ja) 2016-03-01 2022-09-29 優性網膜色素変性の治療のためのａａｖベクター
JP2024168632A JP7872961B2 (ja) 2016-03-01 2024-09-27 優性網膜色素変性の治療のためのａａｖベクター

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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

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JP2019511216A5 JP2019511216A5 (enExample) 2020-04-09
JP7272795B2 true JP7272795B2 (ja) 2023-05-12

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JP2022156496A Active JP7642211B2 (ja) 2016-03-01 2022-09-29 優性網膜色素変性の治療のためのａａｖベクター
JP2024168632A Active JP7872961B2 (ja) 2016-03-01 2024-09-27 優性網膜色素変性の治療のためのａａｖベクター

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JP2024168632A Active JP7872961B2 (ja) 2016-03-01 2024-09-27 優性網膜色素変性の治療のためのａａｖベクター

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EP (2) EP3423582B1 (enExample)
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CN (2) CN115896106A (enExample)
AU (2) AU2017227776C1 (enExample)
CA (1) CA3014671A1 (enExample)
ES (1) ES2923877T3 (enExample)
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WO (1) WO2017151823A1 (enExample)

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IL302455A (en) 2016-03-01 2023-06-01 Univ Florida Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa
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
JP2021522836A (ja) * 2018-05-15 2021-09-02 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法
EP3802836A4 (en) * 2018-06-01 2022-10-19 University of Florida Research Foundation, Incorporated COMPOSITIONS AND METHODS FOR THE TREATMENT OF DOMINANT RETINITIS PIGMENTOSA
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
WO2021030745A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of sca1
CN113038971B (zh) * 2020-04-21 2022-06-24 北京中因科技有限公司 RHO-adRP基于基因编辑的方法和组合物
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
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
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
WO2026087373A1 (en) * 2024-10-21 2026-04-30 MeiraGTx Ocular UK Limited Promoters for driving gene expression in rod photoreceptors and uses thereof

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JP2007530431A (ja) 2004-03-24 2007-11-01 オンコセラピー・サイエンス株式会社 膵臓癌を治療するための組成物および方法
JP2010518821A (ja) 2007-02-16 2010-06-03 ワイス エルエルシー 脂肪細胞の分化を制御する分泌型タンパク質Ｃｃｄｃ８０
CN102061305A (zh) 2010-12-15 2011-05-18 深圳市百恩维生物科技有限公司 一种多ShRNA高效协同沉默基因方法及载体
US20130064815A1 (en) 2011-09-12 2013-03-14 The Trustees Of Princeton University Inducing apoptosis in quiescent cells
US20150159171A1 (en) 2012-05-25 2015-06-11 Commissariat A I'energie Atomique Et Aux Energies Alternatives Vector for the selective silencing of a gene in astrocytes
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IL261014B2 (en) 2023-10-01
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JP2025016451A (ja) 2025-02-04
AU2017227776B2 (en) 2023-07-06
EP3423582B1 (en) 2022-05-04
EP3423582A1 (en) 2019-01-09
JP7872961B2 (ja) 2026-06-11
US20190093111A1 (en) 2019-03-28
ES2923877T3 (es) 2022-10-03
JP2022185052A (ja) 2022-12-13
IL261014B1 (en) 2023-06-01
IL302455A (en) 2023-06-01
AU2023237106B2 (en) 2025-08-28
AU2017227776C1 (en) 2024-01-25
CN109154002B (zh) 2022-08-30
AU2023237106A1 (en) 2023-10-19
US20210324387A1 (en) 2021-10-21
CN109154002A (zh) 2019-01-04
AU2017227776A1 (en) 2018-08-23
JP7642211B2 (ja) 2025-03-10
EP3423582A4 (en) 2019-11-13
JP2019511216A (ja) 2019-04-25
WO2017151823A1 (en) 2017-09-08
US11118185B2 (en) 2021-09-14
IL261014A (en) 2018-10-31
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