CN113271982B - 通过靶向肌营养相关蛋白基因来治疗肌营养不良的方法 - Google Patents

通过靶向肌营养相关蛋白基因来治疗肌营养不良的方法 Download PDF

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CN113271982B
CN113271982B CN201980075173.0A CN201980075173A CN113271982B CN 113271982 B CN113271982 B CN 113271982B CN 201980075173 A CN201980075173 A CN 201980075173A CN 113271982 B CN113271982 B CN 113271982B
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CN113271982A (zh
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吉见英治
吉冈克郎
山形哲也
秦元宝
伊恩·罗伯特·汤普生
尼德西·康纳
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Morris Medical Co ltd
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CN201980075173.0A 2018-11-16 2019-11-15 通过靶向肌营养相关蛋白基因来治疗肌营养不良的方法 Active CN113271982B (zh)

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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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Families Citing this family (12)

* 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.
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
CN117534769A (zh) 2018-04-19 2024-02-09 加利福尼亚大学董事会 用于基因编辑的组合物和方法
MX2020013158A (es) * 2018-06-08 2021-04-29 Modalis Therapeutics Corp Proteina cas9 modificada y uso de la misma.
US20210332094A1 (en) * 2018-08-07 2021-10-28 Modalis Therapeutics Corporation Novel transcription activator
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
US20230272433A1 (en) * 2020-09-29 2023-08-31 Genethon Enhancing Utrophin Expression in Cell by Inducing Mutations Within Utrophin Regulatory Elements and Therapeutic Use Thereof
KR102421411B1 (ko) 2021-10-29 2022-07-18 주식회사 케이에스비튜젠 옥시라세탐을 포함하는 근이영양증 예방 또는 치료용 조성물
US20240050406A1 (en) 2021-04-26 2024-02-15 Ksb Tugen Inc. Composition comprising oxiracetam for preventing or treating muscular disease
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
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
AU2001261063A1 (en) * 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
JP2004535801A (ja) 2001-05-24 2004-12-02 ジェンザイム コーポレイション 筋特異的発現ベクター
ES2258601T3 (es) 2001-11-13 2006-09-01 The Trustees Of The University Of Pennsylvania Un metodo para la identificacion de las secuencias desconocidas del virus adeno-asociado (vaa) y un kit para el metodo.
EP3910063A1 (en) 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
CA2553676A1 (en) 2004-01-28 2005-08-11 Research Development Foundation 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
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
EP2850185A4 (en) * 2012-05-16 2015-12-30 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING UTRN GENE EXPRESSION
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
ES2987399T3 (es) * 2013-06-05 2024-11-14 Univ Duke Edición génica guiada por ARN y regulación génica
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
CA2996982A1 (en) * 2015-09-23 2017-03-30 Universite Laval Modification of the dystrophin gene and uses thereof
US11666666B2 (en) * 2016-02-11 2023-06-06 The Regents Of The University Of California Methods and compositions for modifying a mutant dystrophin gene in a cell's genome
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
Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders;Wojtal D等;《Am J Hum Genet》;第98卷(第01期);第90-101页，特别是第92页右栏第24行-第93页右栏第4行、图1 *

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CO2021006957A2 (es) 2021-06-10
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CN113271982A (zh) 2021-08-17
PH12021551122A1 (en) 2021-11-22
US11473071B2 (en) 2022-10-18
TW202033224A (zh) 2020-09-16
KR20210093954A (ko) 2021-07-28
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AU2019381460A1 (en) 2021-06-17
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