KR20210005889A - Cns 퇴행에 대한 유전자 요법 - Google Patents

Cns 퇴행에 대한 유전자 요법 Download PDF

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KR20210005889A
KR20210005889A KR1020207033123A KR20207033123A KR20210005889A KR 20210005889 A KR20210005889 A KR 20210005889A KR 1020207033123 A KR1020207033123 A KR 1020207033123A KR 20207033123 A KR20207033123 A KR 20207033123A KR 20210005889 A KR20210005889 A KR 20210005889A
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leu
gene
ser
val
gly
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안나히타 케라바라
파벤 바티프로루
라이 프라바카
로데릭 웡
피라팁 프라툼수완
나빈 야라만치
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로켓 파마슈티컬스, 리미티드
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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KR1020207033123A 2018-04-27 2019-04-29 Cns 퇴행에 대한 유전자 요법 Ceased KR20210005889A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862664006P 2018-04-27 2018-04-27
US62/664,006 2018-04-27
PCT/US2019/029744 WO2019210325A1 (en) 2018-04-27 2019-04-29 Gene therapy for cns degeneration

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Publication Number Publication Date
KR20210005889A true KR20210005889A (ko) 2021-01-15

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KR1020207033123A Ceased KR20210005889A (ko) 2018-04-27 2019-04-29 Cns 퇴행에 대한 유전자 요법

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Country Link
US (1) US12378576B2 (https=)
EP (1) EP3810781A4 (https=)
JP (2) JP2021522273A (https=)
KR (1) KR20210005889A (https=)
CN (1) CN112368390B (https=)
AU (1) AU2019261646A1 (https=)
BR (1) BR112020021903A2 (https=)
CA (1) CA3096293A1 (https=)
IL (1) IL278213B2 (https=)
MX (1) MX2020011386A (https=)
RU (1) RU2020134965A (https=)
SG (1) SG11202010061PA (https=)
WO (1) WO2019210325A1 (https=)

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CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
EP3720507A4 (en) * 2017-12-07 2021-08-25 Ramot at Tel-Aviv University Ltd. TREATMENT OF PARKINSON PATIENTS WITH MUTATIONS IN THE LRRK2 GENE
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
JP2023535121A (ja) * 2020-05-20 2023-08-16 スペースクラフト セブン リミテッド ライアビリティ カンパニー 操作されたパーキン及びその使用
JP2023526449A (ja) * 2020-05-21 2023-06-21 ニスノバイオ ジーティー，エルエルシー パーキンソン病を治療するための、増加した活性を有するパーキン変異体の遺伝子治療送達
CA3190720A1 (en) * 2020-08-03 2022-02-10 Prevail Therapeutics, Inc. Aav vectors encoding parkin and uses thereof
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
WO2022040530A2 (en) * 2020-08-21 2022-02-24 Capsida, Inc. Virus compositions for treating mucopolysaccharidosis ii
WO2022066911A1 (en) * 2020-09-23 2022-03-31 Seelos Therapeutics, Inc. Compositions and methods for inhibiting alpha-synuclein aggregation
CN113846122B (zh) * 2021-08-16 2023-08-25 中国医学科学院医学生物学研究所 一种过表达snca的腺相关病毒载体aav-snca、制备方法及其应用
CN115029360B (zh) * 2022-05-30 2024-08-02 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
KR20250142386A (ko) * 2023-02-03 2025-09-30 얀센 파마슈티카 엔브이 파킨슨병에 사용하기 위한 유전자 요법 벡터
WO2025065011A1 (en) * 2023-09-22 2025-03-27 The Regents Of The University Of Colorado, A Body Corporate Gene therapy for diabetic retinopathy
CN119193701B (zh) * 2024-09-13 2025-07-15 暨南大学 帕金森动物模型的构建方法与特异性靶向Parkin基因的gRNA及其应用

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