JP2017512767A - 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 - Google Patents

改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 Download PDF

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JP2017512767A
JP2017512767A JP2016556801A JP2016556801A JP2017512767A JP 2017512767 A JP2017512767 A JP 2017512767A JP 2016556801 A JP2016556801 A JP 2016556801A JP 2016556801 A JP2016556801 A JP 2016556801A JP 2017512767 A JP2017512767 A JP 2017512767A
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exon
dna
seq
sequence
nucleases
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JP2017512767A5 (enExample
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ジャンツ，デレック
スミス，ジェームス，ジェファーソン
ニコルソン，マイケル，ジー．
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プレシジョン バイオサイエンシズ，インク．
プレシジョン バイオサイエンシズ，インク．
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2016556801A 2014-03-12 2015-03-12 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 Pending JP2017512767A (ja)

Applications Claiming Priority (3)

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US201461951648P 2014-03-12 2014-03-12
US61/951,648 2014-03-12
PCT/US2015/020205 WO2015138739A2 (en) 2014-03-12 2015-03-12 Dystrophin gene oxon deletion using engineered nucleases

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JP2019152614A Active JP6832995B2 (ja) 2014-03-12 2019-08-23 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失
JP2021014851A Pending JP2021088563A (ja) 2014-03-12 2021-02-02 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失
JP2022154637A Pending JP2023002555A (ja) 2014-03-12 2022-09-28 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失
JP2024168117A Pending JP2025013784A (ja) 2014-03-12 2024-09-27 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失

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JP2021014851A Pending JP2021088563A (ja) 2014-03-12 2021-02-02 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失
JP2022154637A Pending JP2023002555A (ja) 2014-03-12 2022-09-28 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失
JP2024168117A Pending JP2025013784A (ja) 2014-03-12 2024-09-27 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失

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US (6) US20170106055A1 (enExample)
EP (3) EP4659765A3 (enExample)
JP (5) JP2017512767A (enExample)
AU (3) AU2015229299A1 (enExample)
CA (1) CA2942268A1 (enExample)
DK (1) DK3116533T3 (enExample)
ES (2) ES3037550T3 (enExample)
WO (1) WO2015138739A2 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023549357A (ja) * 2020-11-12 2023-11-24 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
JP7691595B1 (ja) 2024-02-26 2025-06-12 株式会社ニチレイフーズ 欠失した改変ゲノムｄｎａを含む細胞の作製方法、該細胞を含む生物体の作製方法
WO2025182957A1 (ja) * 2024-02-26 2025-09-04 株式会社ニチレイフーズ 改変ｄｎａを含む細胞の作製方法、該細胞を含む生物体の作製方法、及び遺伝子産物の生産方法

Families Citing this family (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
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
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
US20170266320A1 (en) * 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
EP3748004A1 (en) * 2015-04-01 2020-12-09 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
US10603363B2 (en) * 2015-09-08 2020-03-31 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
JP6816133B2 (ja) 2015-10-05 2021-01-20 プレシジョン バイオサイエンシズ，インク． 改変ヒトｔ細胞受容体アルファ定常領域遺伝子を含む遺伝子改変細胞
EP3359660B1 (en) * 2015-10-05 2019-12-04 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
CN113831407B (zh) 2016-05-20 2024-06-11 瑞泽恩制药公司 用于使用多个引导rna来破坏免疫耐受性的方法
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
AU2017364106A1 (en) * 2016-11-28 2019-06-20 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by CRISPR/Cpfl-mediated gene editing
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
EP3617311B1 (en) * 2017-03-30 2024-11-20 Kyoto University Method for inducing exon skipping by genome editing
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
EP3645038B1 (en) 2017-06-30 2026-02-18 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11786554B2 (en) 2018-04-12 2023-10-17 Precision Biosciences, Inc. Optimized engineered nucleases having specificity for the human T cell receptor alpha constant region gene
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12467077B2 (en) * 2018-11-19 2025-11-11 Altius Institute For Biomedical Sciences Compositions and methods for DNA modification detection
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN114026228A (zh) 2019-05-07 2022-02-08 精密生物科学公司 用于识别序列的工程化大范围核酸酶的优化
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
IL296986A (en) * 2020-04-06 2022-12-01 Homology Medicines Inc Adeno-associated virus preparations for ids gene transfer and methods of use thereof
US20230175014A1 (en) * 2020-04-27 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
WO2023108025A1 (en) * 2021-12-08 2023-06-15 Mammoth Biosciences, Inc. Systems and uses thereof for the treatment of dmd-associated diseases

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163628A2 (en) * 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
JP2013544082A (ja) * 2010-10-27 2013-12-12 セレクティス 二本鎖破断−誘導変異誘発の効率を増大させる方法
CA2878654A1 (en) * 2012-07-13 2014-01-16 Association Francaise Contre Les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3202899B1 (en) * 2003-01-28 2020-10-21 Cellectis Custom-made meganuclease and use thereof
ES2582091T3 (es) 2005-10-18 2016-09-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
EP2215223B1 (en) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
EP2313498B1 (en) 2008-07-14 2017-03-15 Precision Biosciences, Inc. Recognition sequences for i-crei-derived meganucleases and uses thereof
EP2480659A2 (en) 2009-09-24 2012-08-01 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
SG169914A1 (en) * 2009-09-29 2011-04-29 Univ Singapore A clinical method for genotyping large genes for mutations that potentially cause disease
CA2799095A1 (en) * 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
EP2714936B1 (en) 2011-06-01 2018-12-12 Precision Biosciences, Inc. Methods and products for producing engineered mammalian cell lines with amplified transgenes
EP2766483B1 (en) * 2011-10-10 2022-03-23 The Hospital For Sick Children Methods and compositions for screening and treating developmental disorders
US8885683B2 (en) * 2011-12-21 2014-11-11 Canon Kabushiki Kaisha Process for forming microstructure of nitride semiconductor, surface emitting laser using two-dimensional photonic crystal and production process thereof
KR102121086B1 (ko) * 2012-11-01 2020-06-09 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
US8697359B1 (en) * 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CA2878645C (en) 2014-01-22 2017-02-21 Alfa Wassermann, Inc. Centrifugation systems with non-contact seal assemblies
AU2016344609B2 (en) * 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
JP7642809B2 (ja) * 2020-11-12 2025-03-10 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013544082A (ja) * 2010-10-27 2013-12-12 セレクティス 二本鎖破断−誘導変異誘発の効率を増大させる方法
WO2013163628A2 (en) * 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
CA2878654A1 (en) * 2012-07-13 2014-01-16 Association Francaise Contre Les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
LI P.ET AL.: "New gene therapy strategies for the deletion of exon 44 of dystrophin gene based on gene editing by", OPEN JOURNAL OF MEDICINAL CHEMISTRY, vol. 3(1), JPN6018046022, March 2013 (2013-03-01), pages 1 - 6, ISSN: 0003925583 *
OUSTEROUT D.G.ET AL.: "Genetic correction of Duchenne muscular dystrophy using zinc finger nucleases", MOLECULAR THERAPY, vol. Vol.21, Supplement 1, JPN6018046020, May 2013 (2013-05-01), pages 111 - 292, ISSN: 0003925582 *
OUSTEROUT D.G.ET AL.: "Reading frame correction by targeted genome editing restores dystrophin expression in cells from duc", MOL THER., vol. 21(9), JPN6018046025, 2013, pages 1718 - 1726, ISSN: 0003925584 *
POPPLEWELL L.ET AL.: "Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in.", HUM GENE THER., vol. 24(7), JPN6018046029, July 2013 (2013-07-01), pages 692 - 701, ISSN: 0003925586 *
ROUSSEAU J.ET AL.: "Endonucleases: tools to correct the dystrophin gene.", J GENE MED., vol. 13(10), JPN6018046027, 2011, pages 522 - 37, ISSN: 0003925585 *

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023549357A (ja) * 2020-11-12 2023-11-24 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
JP7642809B2 (ja) 2020-11-12 2025-03-10 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
JP2025098002A (ja) * 2020-11-12 2025-07-01 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
JP7691595B1 (ja) 2024-02-26 2025-06-12 株式会社ニチレイフーズ 欠失した改変ゲノムｄｎａを含む細胞の作製方法、該細胞を含む生物体の作製方法
WO2025182957A1 (ja) * 2024-02-26 2025-09-04 株式会社ニチレイフーズ 改変ｄｎａを含む細胞の作製方法、該細胞を含む生物体の作製方法、及び遺伝子産物の生産方法
JP2025129511A (ja) * 2024-02-26 2025-09-05 株式会社ニチレイフーズ 欠失した改変ゲノムｄｎａを含む細胞の作製方法、該細胞を含む生物体の作製方法

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