JP2024530050A - 中枢神経系に影響を及ぼす障害の改善された治療のための組成物及び方法 - Google Patents

中枢神経系に影響を及ぼす障害の改善された治療のための組成物及び方法 Download PDF

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JP2024530050A
JP2024530050A JP2024508455A JP2024508455A JP2024530050A JP 2024530050 A JP2024530050 A JP 2024530050A JP 2024508455 A JP2024508455 A JP 2024508455A JP 2024508455 A JP2024508455 A JP 2024508455A JP 2024530050 A JP2024530050 A JP 2024530050A
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pgrn
acid sequence
nucleic acid
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ショー クリストファー
ボク リー ヨウン
ヨン リー ド
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キングス・カレッジ・ロンドン
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    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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JP2024508455A 2021-08-11 2022-08-10 中枢神経系に影響を及ぼす障害の改善された治療のための組成物及び方法 Pending JP2024530050A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163232053P 2021-08-11 2021-08-11
US63/232,053 2021-08-11
US202263331614P 2022-04-15 2022-04-15
US63/331,614 2022-04-15
PCT/EP2022/072487 WO2023017098A2 (en) 2021-08-11 2022-08-10 Compositions and methods for improved treatment of disorders affecting the central nervous system

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JP2024530050A true JP2024530050A (ja) 2024-08-14
JP2024530050A5 JP2024530050A5 (https=) 2025-08-20

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JP2024508455A Pending JP2024530050A (ja) 2021-08-11 2022-08-10 中枢神経系に影響を及ぼす障害の改善された治療のための組成物及び方法

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US (1) US20240358794A1 (https=)
EP (1) EP4384195A2 (https=)
JP (1) JP2024530050A (https=)
AU (1) AU2022327583A1 (https=)
CA (1) CA3228916A1 (https=)
WO (1) WO2023017098A2 (https=)

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CN117286119A (zh) * 2023-09-06 2023-12-26 复旦大学附属中山医院 可静脉注射给药的tpk表达病毒及其治疗阿尔茨海默病的应用

Family Cites Families (18)

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US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DK0752248T3 (da) 1992-11-13 2000-11-13 Idec Pharma Corp Terapeutisk anvendelse af kimæriske og radioaktivt mærkede antistoffer mod humant B-lymfocytbegrænset differentieringsantig
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
DK2311848T3 (da) 2002-12-23 2013-10-14 Vical Inc Kodon-optimerede polynukleotidbaserede vacciner mod human cytomegalovirus-infektion
US7561973B1 (en) 2008-07-31 2009-07-14 Dna Twopointo, Inc. Methods for determining properties that affect an expression property value of polynucleotides in an expression system
US7561972B1 (en) 2008-06-06 2009-07-14 Dna Twopointo, Inc. Synthetic nucleic acids for expression of encoded proteins
US20130090374A1 (en) * 2011-04-20 2013-04-11 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis
JP6028979B2 (ja) 2012-01-30 2016-11-24 日東電工株式会社 核酸固相合成用リンカー及び担体
WO2021016505A1 (en) * 2019-07-24 2021-01-28 Voyager Therapeutics, Inc. Compositions and methods for treating huntington's disease
GB201913974D0 (en) * 2019-09-27 2019-11-13 King S College London Vector

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EP4384195A2 (en) 2024-06-19
WO2023017098A3 (en) 2023-03-23
CA3228916A1 (en) 2023-02-16
AU2022327583A1 (en) 2024-02-22
US20240358794A1 (en) 2024-10-31
WO2023017098A2 (en) 2023-02-16

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