JP7069426B2 - ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 - Google Patents

ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 Download PDF

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JP7069426B2
JP7069426B2 JP2021551108A JP2021551108A JP7069426B2 JP 7069426 B2 JP7069426 B2 JP 7069426B2 JP 2021551108 A JP2021551108 A JP 2021551108A JP 2021551108 A JP2021551108 A JP 2021551108A JP 7069426 B2 JP7069426 B2 JP 7069426B2
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promoter
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base sequence
vector
gene
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JP2022509560A (ja
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英治 吉見
克郎 吉岡
哲也 山形
ユアンバウ シン
イーアン ロバート トンプソン
ニディ カンナ
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Astellas Pharma Inc
Modalis Therapeutics Corp
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JP2021551108A 2018-11-16 2019-11-15 ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 Active JP7069426B2 (ja)

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US201862768474P 2018-11-16 2018-11-16
US62/768,474 2018-11-16
US201962861039P 2019-06-13 2019-06-13
US62/861,039 2019-06-13
US201962931925P 2019-11-07 2019-11-07
US62/931,925 2019-11-07
PCT/JP2019/045716 WO2020101042A1 (en) 2018-11-16 2019-11-15 Method for treating muscular dystrophy by targeting utrophin gene

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102008496B1 (ko) * 2013-03-25 2019-08-07 삼성전자주식회사 단일 센서를 이용하여 기구물의 이동 방향 및 속도를 검출하는 방법 및 장치.

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
AU2019255789B2 (en) 2018-04-19 2025-11-20 The Regents Of The University Of California Compositions and methods for gene editing
JP7412001B2 (ja) * 2018-06-08 2024-01-12 株式会社モダリス 改変されたＣａｓ９タンパク質及びその用途
AU2019317066A1 (en) * 2018-08-07 2021-02-18 Modalis Therapeutics Corporation Novel transcription activator
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
EP4222263A1 (en) 2020-09-29 2023-08-09 Genethon Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements and therapeutic use thereof
US20240050406A1 (en) * 2021-04-26 2024-02-15 Ksb Tugen Inc. Composition comprising oxiracetam for preventing or treating muscular disease
KR102421411B1 (ko) 2021-10-29 2022-07-18 주식회사 케이에스비튜젠 옥시라세탐을 포함하는 근이영양증 예방 또는 치료용 조성물
CN113563428B (zh) * 2021-06-22 2022-08-02 呈诺再生医学科技(珠海横琴新区)有限公司 特定空间结构多肽及其在制备iPSC中的应用
US20230279442A1 (en) * 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
WO2023190935A1 (en) * 2022-03-31 2023-10-05 Modalis Therapeutics Corporation Method for treating myopathies by targeting titin gene
CN116218849B (zh) * 2023-01-18 2024-01-23 昆明理工大学 针对utrophin激活治疗杜氏肌营养不良的sgRNA和应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018169983A1 (en) 2017-03-13 2018-09-20 President And Fellows Of Harvard College Methods of modulating expression of target nucleic acid sequences in a cell

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001083695A2 (en) * 2000-04-28 2001-11-08 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US7557197B2 (en) 2004-01-28 2009-07-07 Oregon Health & Science University Human soluble neuropilin-1 primary polyadenylation signal and uses thereof
ITRM20070523A1 (it) 2007-10-05 2009-04-06 Consiglio Nazionale Ricerche Acido nucleico codificante per una proteina regolatrice specifica della trascrizione dell'utrofina proteina da esso codificata e sue applicazioni
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
CN104540946A (zh) * 2012-05-16 2015-04-22 Rana医疗有限公司 用于调节utrn表达的组合物和方法
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
ITTO20130669A1 (it) * 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
WO2016153012A1 (ja) 2015-03-24 2016-09-29 協和発酵キリン株式会社 核酸含有脂質ナノ粒子
EP3332008A4 (en) * 2015-09-23 2019-01-16 Université Laval CHANGE OF THE DYSTROPHINE GENE AND USES THEREOF
JP6998313B2 (ja) * 2016-02-11 2022-02-04 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 細胞のゲノムにおける変異ジストロフィン遺伝子を修飾する方法及び組成物
US11674128B2 (en) * 2016-12-12 2023-06-13 Tsinghua University Engineering of a minimal SaCas9 CRISPR/Cas system for gene editing and transcriptional regulation optimized by enhanced guide RNA
EP3579858A4 (en) * 2017-02-07 2020-12-23 The Regents of The University of California GENE THERAPY AGAINST HAPLOINSUFFICIENCY
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
CA3097375A1 (en) 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
WO2019236081A1 (en) * 2018-06-06 2019-12-12 Salk Institute For Biological Studies Targeted gene activation using modified guide rna

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018169983A1 (en) 2017-03-13 2018-09-20 President And Fellows Of Harvard College Methods of modulating expression of target nucleic acid sequences in a cell

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
The American Journal of Human Genetics，2016年，Vol.98，PP.90-101

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102008496B1 (ko) * 2013-03-25 2019-08-07 삼성전자주식회사 단일 센서를 이용하여 기구물의 이동 방향 및 속도를 검출하는 방법 및 장치.

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TW202033224A (zh) 2020-09-16
IL283123B2 (en) 2026-03-01
SG11202105030VA (en) 2021-06-29
JP2022115976A (ja) 2022-08-09
WO2020101042A1 (en) 2020-05-22
CN113271982A (zh) 2021-08-17
CN113271982B (zh) 2024-07-30
CA3119618A1 (en) 2020-05-22
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PH12021551122A1 (en) 2021-11-22
US20210355464A1 (en) 2021-11-18
IL283123A (en) 2021-06-30
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WO2020101042A8 (en) 2020-07-30
EP3880255A1 (en) 2021-09-22
CO2021006957A2 (es) 2021-06-10
US11473071B2 (en) 2022-10-18
KR20210093954A (ko) 2021-07-28

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