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)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Biophysics (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)

Application Number Priority Date Filing Date Title
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) EP3858376B1 (enExample)
JP (5) JP2017512767A (enExample)
AU (3) AU2015229299A1 (enExample)
CA (1) CA2942268A1 (enExample)
DK (1) DK3116533T3 (enExample)
ES (2) ES2821149T3 (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
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
SMT202100516T1 (it) 2013-04-16 2021-11-12 Regeneron Pharma Modificazione mirata del genoma di ratto
US20150044192A1 (en) 2013-08-09 2015-02-12 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
ES2670983T3 (es) 2013-11-07 2018-06-04 Editas Medicine, Inc. Métodos y composiciones relacionados con CRISPR con ARNg rectores
BR112016013400B1 (pt) 2013-12-11 2023-02-14 Regeneron Pharmaceuticals, Inc. Método in vitro para modificar um genoma em um lócus genômico de interesse em uma célula pluripotente
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
MX389266B (es) 2014-11-21 2025-03-20 Regeneron Pharma Metodos y composiciones para la modificacion genetica dirigida mediante el uso de arn guia combinados.
WO2016089866A1 (en) * 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
AU2016244033A1 (en) * 2015-04-01 2017-10-19 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
EP3680329B1 (en) * 2015-10-05 2021-06-09 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
AU2016333898B2 (en) 2015-10-05 2020-11-12 Precision Biosciences, Inc. Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
WO2017201476A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
US20200046854A1 (en) * 2016-11-28 2020-02-13 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
US10745677B2 (en) 2016-12-23 2020-08-18 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
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
JP7584777B2 (ja) * 2017-03-30 2024-11-18 国立大学法人京都大学 ゲノム編集によるエクソンスキッピング誘導方法
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
US11053484B2 (en) 2017-06-30 2021-07-06 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
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
IL317539A (en) 2018-04-12 2025-02-01 Prec Biosciences Inc Optimized engineered nucleases with specificity for the human T-cell receptor alpha constant region gene
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
US12281338B2 (en) 2018-10-29 2025-04-22 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
CA3130488A1 (en) 2019-03-19 2020-09-24 David R. Liu 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
CA3137975A1 (en) 2019-05-07 2020-11-12 Precision Biosciences, Inc. Optimization of engineered meganucleases for recognition sequences
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
KR20220164743A (ko) * 2020-04-06 2022-12-13 호몰로지 메디슨, 인크. Ids 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
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
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2021231259A1 (en) 2020-05-11 2021-11-18 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
AU2004208031B2 (en) * 2003-01-28 2009-10-08 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application 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
WO2009059195A2 (en) 2007-10-31 2009-05-07 Precision Biosciences 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)
AU2009271011B2 (en) 2008-07-14 2015-10-22 Precision Biosciences, Inc. Recognition sequences for I-Crei-derived meganucleases and uses thereof
US8802437B2 (en) * 2009-09-24 2014-08-12 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
US10221454B2 (en) * 2011-10-10 2019-03-05 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
AU2013337651B2 (en) * 2012-11-01 2018-12-13 Factor Bioscience Inc. Methods and products for expressing proteins in cells
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
EP4632068A3 (en) * 2015-10-28 2026-01-21 Vertex Pharmaceuticals Inc. Materials and methods for treatment of duchenne muscular dystrophy
KR20250099412A (ko) * 2020-11-12 2025-07-01 프리시젼 바이오사이언시스 인코포레이티드 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제

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 *

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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
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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