TW202033224A - 藉靶向肌營養相關蛋白基因治療肌肉萎縮症之方法 - Google Patents

藉靶向肌營養相關蛋白基因治療肌肉萎縮症之方法 Download PDF

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TW202033224A
TW202033224A TW108141671A TW108141671A TW202033224A TW 202033224 A TW202033224 A TW 202033224A TW 108141671 A TW108141671 A TW 108141671A TW 108141671 A TW108141671 A TW 108141671A TW 202033224 A TW202033224 A TW 202033224A
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seq
polynucleotide
sequence
base sequence
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TW108141671A
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吉見英治
吉岡克郎
山形哲也
元寶 秦
伊恩 羅伯特 湯普生
尼德西 康納
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日商安斯泰來製藥股份有限公司
日商摩大力斯醫療公司
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TW108141671A 2018-11-16 2019-11-15 藉靶向肌營養相關蛋白基因治療肌肉萎縮症之方法 TW202033224A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
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

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US (1) US11473071B2 (enExample)
EP (1) EP3880255A1 (enExample)
JP (2) JP7069426B2 (enExample)
KR (1) KR20210093954A (enExample)
CN (1) CN113271982B (enExample)
AU (1) AU2019381460A1 (enExample)
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Families Citing this family (12)

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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
CA3103088A1 (en) * 2018-06-08 2019-12-12 Modalis Therapeutics Corporation Modified cas9 protein and use thereof
EP3833758A4 (en) * 2018-08-07 2022-05-18 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
CN116323941A (zh) * 2020-09-29 2023-06-23 吉尼松公司 通过诱导抗肌萎缩蛋白相关蛋白调节元件内的突变增强细胞中抗肌萎缩蛋白相关蛋白表达及其治疗用途
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
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

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2407309C (en) * 2000-04-28 2011-08-02 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
EP2017338A1 (en) 2001-05-24 2009-01-21 Genzyme Corporation Muscle-specific expression vectors
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
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
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
KR20160074368A (ko) * 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
SI3401400T1 (sl) 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
KR20230136697A (ko) * 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
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
KR20170130531A (ko) 2015-03-24 2017-11-28 교와 핫꼬 기린 가부시키가이샤 핵산 함유 지질 나노 입자
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
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
WO2018204764A1 (en) * 2017-05-05 2018-11-08 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

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US11473071B2 (en) 2022-10-18
JP7069426B2 (ja) 2022-05-17
WO2020101042A1 (en) 2020-05-22
SG11202105030VA (en) 2021-06-29
JP2022115976A (ja) 2022-08-09
CN113271982B (zh) 2024-07-30
MX2021005720A (es) 2021-07-21
AU2019381460A1 (en) 2021-06-17
PH12021551122A1 (en) 2021-11-22
KR20210093954A (ko) 2021-07-28
WO2020101042A8 (en) 2020-07-30
CA3119618A1 (en) 2020-05-22
BR112021009481A2 (pt) 2021-08-17
JP2022509560A (ja) 2022-01-20
IL283123A (en) 2021-06-30
IL283123B1 (en) 2025-11-01
EP3880255A1 (en) 2021-09-22
CN113271982A (zh) 2021-08-17
CO2021006957A2 (es) 2021-06-10
US20210355464A1 (en) 2021-11-18

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