JP2019536782A5 - - Google Patents

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JP2019536782A5
JP2019536782A5 JP2019528478A JP2019528478A JP2019536782A5 JP 2019536782 A5 JP2019536782 A5 JP 2019536782A5 JP 2019528478 A JP2019528478 A JP 2019528478A JP 2019528478 A JP2019528478 A JP 2019528478A JP 2019536782 A5 JP2019536782 A5 JP 2019536782A5
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Japan
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promoter
composition according
composition
sequence encoding
present
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JP2019528478A
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JP2019536782A (ja
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Priority claimed from PCT/US2017/063468 external-priority patent/WO2018098480A1/en
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JP2019528478A 2016-11-28 2017-11-28 CRISPR/Cpf1媒介性遺伝子編集による筋ジストロフィーの予防 Pending JP2019536782A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662426853P 2016-11-28 2016-11-28
US62/426,853 2016-11-28
PCT/US2017/063468 WO2018098480A1 (en) 2016-11-28 2017-11-28 Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing

Publications (2)

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JP2019536782A JP2019536782A (ja) 2019-12-19
JP2019536782A5 true JP2019536782A5 (enExample) 2021-01-14

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JP2019528478A Pending JP2019536782A (ja) 2016-11-28 2017-11-28 CRISPR/Cpf1媒介性遺伝子編集による筋ジストロフィーの予防

Country Status (8)

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US (1) US20200046854A1 (enExample)
EP (1) EP3545090A1 (enExample)
JP (1) JP2019536782A (enExample)
CN (1) CN110382695A (enExample)
AU (1) AU2017364106A1 (enExample)
CA (1) CA3044531A1 (enExample)
MX (1) MX2019006157A (enExample)
WO (1) WO2018098480A1 (enExample)

Families Citing this family (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP4036228A1 (en) 2015-11-13 2022-08-03 Avellino Lab USA, Inc. Methods for the treatment of corneal dystrophies
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
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
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
WO2020225754A1 (en) * 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
CN110892069B (zh) * 2017-03-30 2024-02-09 国立大学法人京都大学 基于基因组编辑的外显子跳跃诱导方法
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020122104A1 (ja) * 2018-12-11 2020-06-18 国立大学法人京都大学 ゲノムdnaに欠失を誘導する方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20220145305A1 (en) * 2019-02-12 2022-05-12 Universita' Degli Studi Di Trento CAS12a GUIDE RNA MOLECULES AND USES THEREOF
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
JP7881154B2 (ja) * 2019-04-12 2026-06-29 デューク ユニバーシティ ジストロフィン機能を修復するためのCRISPR/Casをベースにした塩基編集組成物
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
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110499333A (zh) * 2019-08-01 2019-11-26 广州德赫生物科技有限公司 用于修复dmd基因突变的核酸序列及系统
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021178474A1 (en) * 2020-03-04 2021-09-10 Regeneron Pharmaceuticals, Inc. A rodent model of b4galt1-mediated functions
US20240287486A1 (en) * 2020-04-24 2024-08-29 University Of Massachusetts Improved cas 12a/nls mediated therapeutic gene editing platforms
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
EP4149622A4 (en) * 2020-05-12 2024-12-18 Myogene Bio LLC IMMUNOSUPPRESSIVE AGENTS AND METHODS OF RE-DOSING VIRAL ADMINISTRATION FOR GENE THERAPY
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN116529365A (zh) * 2020-09-09 2023-08-01 弗特克斯药品有限公司 用于治疗杜氏肌营养不良的组合物和方法
US20220096606A1 (en) * 2020-09-09 2022-03-31 Vertex Pharmaceuticals Incorporated Compositions and Methods for Treatment of Duchenne Muscular Dystrophy
WO2022140340A1 (en) * 2020-12-22 2022-06-30 Vertex Pharmaceuticals Incorporated Compositions comprising an rna guide targeting dmd and uses thereof
WO2022251181A1 (en) * 2021-05-25 2022-12-01 The Board Of Regents Of The University Of Texas System Correction of duchenne muscular dystrophy mutations with all-in-one adeno-associated virus-delivered single-cut crispr
CN118591391A (zh) * 2021-07-09 2024-09-03 达因疗法公司 肌肉靶向复合物及其用于治疗肌养蛋白病的用途
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
CN114214360A (zh) * 2021-12-27 2022-03-22 西安英创生物技术有限公司 先天性肌无力小鼠模型、其构建方法及应用
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
TW202345911A (zh) * 2022-03-08 2023-12-01 美商維泰克斯製藥公司 用於治療杜興氏肌肉失養症(duchenne muscular dystrophy)之部分外顯子44、50及53之精確切除
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
CN120648680A (zh) * 2024-03-15 2025-09-16 迦进生物医药(上海)有限公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的药物
CN118086408A (zh) * 2024-04-25 2024-05-28 华南理工大学 一种可实现靶向肌肉细胞基因编辑的仿病毒颗粒及其制备方法和应用

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK99653C (da) 1959-06-24 1964-08-31 Skaanska Aettikfabriken Ab Pynteliste til brug som sammenfojningsliste, hjørneliste eller endeliste ved montering af beklædningsplader.
EP0273085A1 (en) 1986-12-29 1988-07-06 IntraCel Corporation A method for internalizing nucleic acids into eukaryotic cells
EP3133160B1 (en) * 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Exon skipping compositions for dmd
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
HK1220154A1 (zh) * 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
AU2015229299A1 (en) * 2014-03-12 2016-09-22 Precision Biosciences, Inc. Dystrophin gene exon deletion using engineered nucleases
CA2957932A1 (en) * 2014-08-11 2016-02-18 The Board Of Trustees Of The University Of Illinois Devices and related methods for epidermal characterization of biofluids
US20160058889A1 (en) * 2014-08-11 2016-03-03 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cas9-mediated gene editing
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
KR20240132120A (ko) * 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
EP3332008A4 (en) * 2015-09-23 2019-01-16 Université Laval CHANGE OF THE DYSTROPHINE GENE AND USES THEREOF
EP4279084B1 (en) * 2015-10-28 2025-06-11 Vertex Pharmaceuticals Inc. Materials and methods for treatment of duchenne muscular dystrophy
JP7490211B2 (ja) * 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用

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