JP2025039642A5 - - Google Patents

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Publication number
JP2025039642A5
JP2025039642A5 JP2025000101A JP2025000101A JP2025039642A5 JP 2025039642 A5 JP2025039642 A5 JP 2025039642A5 JP 2025000101 A JP2025000101 A JP 2025000101A JP 2025000101 A JP2025000101 A JP 2025000101A JP 2025039642 A5 JP2025039642 A5 JP 2025039642A5
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JP
Japan
Prior art keywords
disease
protein
cells
vector
subject
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2025000101A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025039642A (ja
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Publication date
Priority claimed from JP2019550825A external-priority patent/JP7233375B2/ja
Application filed filed Critical
Publication of JP2025039642A publication Critical patent/JP2025039642A/ja
Publication of JP2025039642A5 publication Critical patent/JP2025039642A5/ja
Pending legal-status Critical Current

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JP2025000101A 2017-03-15 2025-01-06 リソソーム障害を治療する方法 Pending JP2025039642A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762471741P 2017-03-15 2017-03-15
US62/471,741 2017-03-15
US201762507713P 2017-05-17 2017-05-17
US62/507,713 2017-05-17
JP2019550825A JP7233375B2 (ja) 2017-03-15 2018-03-15 リソソーム障害を治療する方法
PCT/US2018/022598 WO2018170239A1 (en) 2017-03-15 2018-03-15 Methods of treating lysosomal disorders
JP2023005909A JP2023052475A (ja) 2017-03-15 2023-01-18 リソソーム障害を治療する方法

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2023005909A Division JP2023052475A (ja) 2017-03-15 2023-01-18 リソソーム障害を治療する方法

Publications (2)

Publication Number Publication Date
JP2025039642A JP2025039642A (ja) 2025-03-21
JP2025039642A5 true JP2025039642A5 (https=) 2026-02-17

Family

ID=63523990

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019550825A Active JP7233375B2 (ja) 2017-03-15 2018-03-15 リソソーム障害を治療する方法
JP2023005909A Pending JP2023052475A (ja) 2017-03-15 2023-01-18 リソソーム障害を治療する方法
JP2025000101A Pending JP2025039642A (ja) 2017-03-15 2025-01-06 リソソーム障害を治療する方法

Family Applications Before (2)

Application Number Title Priority Date Filing Date
JP2019550825A Active JP7233375B2 (ja) 2017-03-15 2018-03-15 リソソーム障害を治療する方法
JP2023005909A Pending JP2023052475A (ja) 2017-03-15 2023-01-18 リソソーム障害を治療する方法

Country Status (8)

Country Link
US (2) US11806367B2 (https=)
EP (1) EP3596111A4 (https=)
JP (3) JP7233375B2 (https=)
CN (1) CN110418800A (https=)
AU (2) AU2018236353B2 (https=)
CA (1) CA3056617A1 (https=)
IL (1) IL269082A (https=)
WO (1) WO2018170239A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3056617A1 (en) 2017-03-15 2018-09-20 The Regents Of The University Of California Methods of treating lysosomal disorders
KR102918544B1 (ko) 2018-07-12 2026-01-27 로켓 파마슈티컬스, 리미티드 다논병을 치료하기 위한 유전자 요법 벡터
CN109880849B (zh) * 2019-01-03 2023-04-11 四川大学 靶向肿瘤相关巨噬细胞的GHOST-shRNA表达载体复合物及其应用
WO2020160458A1 (en) 2019-02-01 2020-08-06 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
WO2020179931A1 (ja) * 2019-03-07 2020-09-10 国立大学法人 東京医科歯科大学 ハイスループット遺伝子編集技術
US12319928B2 (en) * 2019-03-21 2025-06-03 The Regents Of The University Of Colorado, A Body Corporate Methods of treating Danon disease
WO2020210218A1 (en) * 2019-04-12 2020-10-15 The Regents Of The University Of California Methods for treating inherited eye defects
WO2021073607A1 (en) * 2019-10-17 2021-04-22 Westlake Therapeutics (Hangzhou) Co. Limited Engineering red blood cells for treatment of lysosomal storage diseases
GB202003536D0 (en) * 2020-03-11 2020-04-29 Ucl Business Ltd Gene therapy
US12173304B2 (en) * 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
CN114934066B (zh) * 2022-04-28 2024-10-18 深圳临研医学有限公司 石骨症的基因编辑体系及其应用
CN118388623B (zh) * 2024-06-28 2024-10-08 首都医科大学 Sialin蛋白在调控细胞线粒体功能中的应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3196301B1 (en) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
JP2015529466A (ja) 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター
HK1208371A1 (en) 2012-10-09 2016-03-04 桑比欧公司 Methods and compositions for treatment of retinal degeneration
US10155794B2 (en) 2013-07-16 2018-12-18 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to CEP290
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
PE20180772A1 (es) * 2015-07-01 2018-05-07 Univ California Vectores retrovirales que contienen un promotor de ubiquitina c humana de orientacion inversa
US10670862B2 (en) 2015-07-02 2020-06-02 Microsoft Technology Licensing, Llc Diffractive optical elements with asymmetric profiles
JP7089282B2 (ja) * 2015-07-20 2022-06-22 アンジオクライン・バイオサイエンス・インコーポレイテッド 幹細胞移植のための方法および組成物
CA3056617A1 (en) 2017-03-15 2018-09-20 The Regents Of The University Of California Methods of treating lysosomal disorders

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