JP2023551873A - Ｃａｇリピート病を処置するためのｒｎａ標的化組成物および方法 - Google Patents

Ｃａｇリピート病を処置するためのｒｎａ標的化組成物および方法 Download PDF

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JP2023551873A
JP2023551873A JP2023533276A JP2023533276A JP2023551873A JP 2023551873 A JP2023551873 A JP 2023551873A JP 2023533276 A JP2023533276 A JP 2023533276A JP 2023533276 A JP2023533276 A JP 2023533276A JP 2023551873 A JP2023551873 A JP 2023551873A
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sequence
seq
rna
cag
protein
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デイビッド エー． ネルズ，
ランジャン バトラ，
ダニエラ ロス，
ディミトリオス ジソーリス，
アンジェリーン タ，
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ロックアネイビオ， インコーポレイテッド
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JP2023533276A 2020-12-01 2021-12-01 Ｃａｇリピート病を処置するためのｒｎａ標的化組成物および方法 Pending JP2023551873A (ja)

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US202063119977P 2020-12-01 2020-12-01
US63/119,977 2020-12-01
US202063130060P 2020-12-23 2020-12-23
US63/130,060 2020-12-23
PCT/US2021/061482 WO2022119974A1 (en) 2020-12-01 2021-12-01 Rna-targeting compositions and methods for treating cag repeat diseases

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JP2023551873A true JP2023551873A (ja) 2023-12-13
JPWO2022119974A5 JPWO2022119974A5 (https=) 2024-10-29
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US (1) US20240000972A1 (https=)
EP (1) EP4255470A1 (https=)
JP (1) JP2023551873A (https=)
KR (1) KR20230127221A9 (https=)
AU (1) AU2021391643A1 (https=)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023551874A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド 筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
WO2022221278A1 (en) * 2021-04-12 2022-10-20 Locanabio, Inc. Compositions and methods comprising hybrid promoters
JP2025525378A (ja) * 2022-06-21 2025-08-05 リーゲル セラピューティクス，インコーポレイティッド 遺伝子調節エレメント及びその使用
CN119320798A (zh) * 2023-07-17 2025-01-17 中国科学院上海营养与健康研究所 一种可用于筛选针对rna重复序列药物的报告系统
WO2025155722A1 (en) * 2024-01-16 2025-07-24 Astellas Gene Therapies, Inc. Muscle selective hybrid regulatory combinations and methods of use thereof for the treatment of myotonic dystrophy type 1

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019236982A1 (en) * 2018-06-08 2019-12-12 Locana, Inc. Rna-targeting fusion protein compositions and methods for use
JP2023551874A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド 筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
CN101895633A (zh) 2010-07-14 2010-11-24 中兴通讯股份有限公司 一种移动终端及其解锁方法
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
WO2013058404A1 (ja) 2011-10-21 2013-04-25 国立大学法人九州大学 Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用
US10330674B2 (en) 2015-01-13 2019-06-25 Massachusetts Institute Of Technology Pumilio domain-based modular protein architecture for RNA binding
US10876101B2 (en) 2017-03-28 2020-12-29 Locanabio, Inc. CRISPR-associated (Cas) protein
US11168322B2 (en) 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US10476825B2 (en) 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019236982A1 (en) * 2018-06-08 2019-12-12 Locana, Inc. Rna-targeting fusion protein compositions and methods for use
JP2023551874A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド 筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023551874A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド 筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法

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EP4255470A1 (en) 2023-10-11
KR20230127221A (ko) 2023-08-31
CA3200453A1 (en) 2022-06-09
US20240000972A1 (en) 2024-01-04
WO2022119974A1 (en) 2022-06-09
KR20230127221A9 (ko) 2024-12-06
AU2021391643A9 (en) 2023-08-17

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