JP2022545378A - ＳＣＡ１の治療のための、導入遺伝子およびイントロン由来ｍｉＲＮＡを組み合わせた治療法 - Google Patents

ＳＣＡ１の治療のための、導入遺伝子およびイントロン由来ｍｉＲＮＡを組み合わせた治療法 Download PDF

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JP2022545378A
JP2022545378A JP2022509153A JP2022509153A JP2022545378A JP 2022545378 A JP2022545378 A JP 2022545378A JP 2022509153 A JP2022509153 A JP 2022509153A JP 2022509153 A JP2022509153 A JP 2022509153A JP 2022545378 A JP2022545378 A JP 2022545378A
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aav
promoter
nucleic acid
sequence
expression cassette
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ビヴァリー エル． ダヴィッドソン
エリー カレル
アレハンドロ マス モンティス
ミーガン エス． カイザー
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Childrens Hospital of Philadelphia CHOP
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JP2022509153A 2019-08-15 2020-08-14 ＳＣＡ１の治療のための、導入遺伝子およびイントロン由来ｍｉＲＮＡを組み合わせた治療法 Pending JP2022545378A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962887209P 2019-08-15 2019-08-15
US62/887,209 2019-08-15
PCT/US2020/046499 WO2021030745A1 (en) 2019-08-15 2020-08-14 Combined transgene and intron-derived mirna therapy for treatment of sca1

Publications (2)

Publication Number Publication Date
JP2022545378A true JP2022545378A (ja) 2022-10-27
JP2022545378A5 JP2022545378A5 (https=) 2023-08-15

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JP2022509153A Pending JP2022545378A (ja) 2019-08-15 2020-08-14 ＳＣＡ１の治療のための、導入遺伝子およびイントロン由来ｍｉＲＮＡを組み合わせた治療法

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US (1) US20220288101A1 (https=)
EP (1) EP4013414A4 (https=)
JP (1) JP2022545378A (https=)
CN (1) CN114555084A (https=)
AU (1) AU2020330108A1 (https=)
BR (1) BR112022002794A2 (https=)
CA (1) CA3151122A1 (https=)
MX (1) MX2022001984A (https=)
WO (1) WO2021030745A1 (https=)

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US11008627B2 (en) 2019-08-15 2021-05-18 Talis Biomedical Corporation Diagnostic system
WO2024044469A1 (en) * 2022-08-26 2024-02-29 The Children's Hospital Of Philadelphia Mirnas targeting atnx2 for the treatment of als and sca2
EP4651883A1 (en) * 2023-01-19 2025-11-26 uniQure biopharma B.V. Pharmaceutical formulations of gene delivery vehicles

Citations (3)

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Publication number Priority date Publication date Assignee Title
JP2014511180A (ja) * 2011-02-22 2014-05-15 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
JP2017509632A (ja) * 2014-03-21 2017-04-06 ジェンザイム・コーポレーション 網膜色素変性症のための遺伝子治療
JP2019511216A (ja) * 2016-03-01 2019-04-25 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 優性網膜色素変性の治療のためのａａｖベクター

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US7001733B1 (en) * 1998-05-12 2006-02-21 Rigel Pharmaceuticals, Inc. Methods and compositions for screening for modulations of IgE synthesis, secretion and switch rearrangement
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
DK2164967T3 (en) * 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
EP3146051B8 (en) * 2014-05-20 2019-11-27 University of Iowa Research Foundation Huntington's disease therapeutic compounds
MA40782A (fr) * 2014-10-03 2017-08-08 Hocuslocus Llc Procédés et compositions utilisant un arn non codant en vue de la culture et de la sélection de cellules
EP3261582B1 (en) * 2015-02-26 2021-01-06 Remodeless CV Ltd. Methods and compositions relating to leptin antagonists
WO2016196507A1 (en) * 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
KR20200119782A (ko) * 2017-12-06 2020-10-20 오비드 테라퓨틱스 인크. 발작 장애 치료 시 mir101 또는 mir128의 용도
US20210355509A1 (en) * 2018-08-07 2021-11-18 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods

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Publication number Priority date Publication date Assignee Title
JP2014511180A (ja) * 2011-02-22 2014-05-15 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
JP2017509632A (ja) * 2014-03-21 2017-04-06 ジェンザイム・コーポレーション 網膜色素変性症のための遺伝子治療
JP2019511216A (ja) * 2016-03-01 2019-04-25 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 優性網膜色素変性の治療のためのａａｖベクター

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MEGAN S. KEISER ET AL.: ""RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1"", NEUROBIOLOGY OF DISEASE, vol. 56, JPN6024028932, August 2013 (2013-08-01), pages 6 - 13, ISSN: 0005732607 *

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Publication number Publication date
WO2021030745A1 (en) 2021-02-18
CN114555084A (zh) 2022-05-27
EP4013414A4 (en) 2023-09-27
EP4013414A1 (en) 2022-06-22
MX2022001984A (es) 2022-05-19
US20220288101A1 (en) 2022-09-15
BR112022002794A2 (pt) 2022-08-09
CA3151122A1 (en) 2021-02-18
AU2020330108A1 (en) 2022-03-17

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