CN110198712A - 用于治疗神经退行性疾病的基因转移组合物、方法和用途 - Google Patents

用于治疗神经退行性疾病的基因转移组合物、方法和用途 Download PDF

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Publication number
CN110198712A
CN110198712A CN201780067919.4A CN201780067919A CN110198712A CN 110198712 A CN110198712 A CN 110198712A CN 201780067919 A CN201780067919 A CN 201780067919A CN 110198712 A CN110198712 A CN 110198712A
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aav
mammal
tpp1
particles
aav particles
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Chinese (zh)
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B·L·戴维森
Y·H·陈
L·特塞多尔
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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201780067919.4A 2016-11-04 2017-11-03 用于治疗神经退行性疾病的基因转移组合物、方法和用途 Pending CN110198712A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662418033P 2016-11-04 2016-11-04
US62/418033 2016-11-04
PCT/US2017/059986 WO2018085688A1 (en) 2016-11-04 2017-11-03 Gene transfer compositions, methods and uses for treating neurodegenerative diseases

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CN110198712A true CN110198712A (zh) 2019-09-03

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US (1) US20190269797A1 (enExample)
EP (1) EP3534892A4 (enExample)
JP (2) JP2019537576A (enExample)
CN (1) CN110198712A (enExample)
AU (1) AU2017355502B2 (enExample)
BR (1) BR112019009074A2 (enExample)
CA (1) CA3041548A1 (enExample)
MX (1) MX2019005266A (enExample)
WO (1) WO2018085688A1 (enExample)

Cited By (1)

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CN115244181B (zh) * 2020-03-11 2025-08-08 上海信致医药科技有限公司 阿司匹林化合物在增加核酸表达中的新型用途

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US9818239B2 (en) 2015-08-20 2017-11-14 Zendrive, Inc. Method for smartphone-based accident detection
CN110809583A (zh) 2017-06-07 2020-02-18 瑞泽恩制药公司 用于内化酶的组合物和方法
EP3698340A4 (en) 2017-10-20 2021-07-14 Zendrive, Inc. PROCEDURE AND SYSTEM FOR VEHICLE ASSOCIATED COMMUNICATION
KR20200118151A (ko) 2018-02-07 2020-10-14 리제너론 파마슈티칼스 인코포레이티드 치료 단백질 전달을 위한 방법 및 조성물
MX2020012350A (es) 2018-05-17 2021-01-29 Regeneron Pharma Anticuerpos anti-cd63, conjugados y usos de estos.
KR20210084459A (ko) 2018-09-26 2021-07-07 캘리포니아 인스티튜트 오브 테크놀로지 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물
BR112021009370A2 (pt) * 2018-11-14 2021-08-17 Regenxbio Inc. método de tratamento da doença de batten cln2, composição farmacêutica e kit
WO2020160486A1 (en) * 2019-02-01 2020-08-06 Spark Therapeutics, Inc. Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2
JP2023504269A (ja) 2019-12-03 2023-02-02 ゼンドライヴ，インコーポレイテッド ルートのリスク判定方法およびシステム
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain

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Publication number Priority date Publication date Assignee Title
CN115244181B (zh) * 2020-03-11 2025-08-08 上海信致医药科技有限公司 阿司匹林化合物在增加核酸表达中的新型用途

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AU2017355502B2 (en) 2023-08-31
US20190269797A1 (en) 2019-09-05
RU2019117062A3 (enExample) 2021-03-11
EP3534892A4 (en) 2020-05-27
JP2019537576A (ja) 2019-12-26
CA3041548A1 (en) 2018-05-11
JP2023002721A (ja) 2023-01-10
BR112019009074A2 (pt) 2019-07-16
EP3534892A1 (en) 2019-09-11
WO2018085688A1 (en) 2018-05-11
AU2017355502A1 (en) 2019-05-16
RU2019117062A (ru) 2020-12-04
MX2019005266A (es) 2019-09-27

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