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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  • 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)
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  • 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)

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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) EP3116533B1 (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 (2)

* 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

Families Citing this family (51)

* 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
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
EP4372090A3 (en) 2013-11-07 2024-08-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
CN110951779B (zh) 2013-12-11 2024-04-16 瑞泽恩制药公司 用于靶向修饰基因组的方法和组合物
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
BR112017010547A2 (pt) 2014-11-21 2018-02-27 Regeneron Pharma métodos para produção de uma modificação bialélica, modificação de um genoma dentro de uma célula, produção de uma geração f0 de um animal não humano e identificação de uma inserção de um inserto de ácido nucleico.
WO2016089866A1 (en) * 2014-12-01 2016-06-09 President And Fellows Of Harvard College Rna-guided systems for in vivo gene editing
WO2016161380A1 (en) * 2015-04-01 2016-10-06 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
EP4530354A3 (en) * 2015-09-08 2025-09-10 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
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
ES2768984T3 (es) 2015-10-05 2020-06-24 Prec Biosciences Inc Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
IL262888B1 (en) 2016-05-20 2025-10-01 Regeneron Pharma 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
US11661590B2 (en) 2016-08-09 2023-05-30 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
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
WO2018098480A1 (en) * 2016-11-28 2018-05-31 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
EP3592381A1 (en) 2017-03-09 2020-01-15 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
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
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
AU2017407272B2 (en) * 2017-03-30 2024-06-13 Kyoto University Method for inducing exon skipping by genome editing
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CA3068465A1 (en) 2017-06-30 2019-01-03 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
US11732274B2 (en) 2017-07-28 2023-08-22 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
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
KR102617818B1 (ko) 2018-04-12 2023-12-27 프리시젼 바이오사이언시스 인코포레이티드 인간 t 세포 수용체 알파 불변 영역 유전자에 대한 특이성을 갖는 최적화된 조작된 뉴클레아제
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
EP3884063B1 (en) * 2018-11-19 2023-11-01 Altius Institute For Biomedical Sciences Compositions and methods for detection of cleavage of genomic dna by a nuclease
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
WO2020214842A1 (en) 2019-04-17 2020-10-22 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
WO2021072328A1 (en) 2019-10-10 2021-04-15 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
EP4143308A4 (en) * 2020-04-27 2024-06-26 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR REDUCING NUCLEASE EXPRESSION AND OFF-TARGET ACTIVITY USING A PROMOTER WITH LOW TRANSCRIPTION ACTIVITY
GB2614813B (en) 2020-05-08 2025-05-07 Harvard College 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
EP4444886A1 (en) * 2021-12-08 2024-10-16 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
JP2006518372A (ja) * 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用
US8021867B2 (en) 2005-10-18 2011-09-20 Duke University Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity
EP3578646A3 (en) 2007-10-31 2020-03-18 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)
ES2625941T3 (es) 2008-07-14 2017-07-21 Precision Biosciences, Inc. Secuencias de reconocimiento para meganucleasas derivadas de i-crei y sus usos
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
DK2714936T3 (en) * 2011-06-01 2019-03-25 Prec Biosciences Inc METHODS AND PRODUCTS FOR PRODUCING MANIPULATED MAMMAL CELL LINES WITH AMPLIFIED TRANSGENES
CA2851388C (en) * 2011-10-10 2023-11-21 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
WO2014071219A1 (en) * 2012-11-01 2014-05-08 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
EP4279084B1 (en) * 2015-10-28 2025-06-11 Vertex Pharmaceuticals Inc. Materials and methods for treatment of duchenne muscular dystrophy
EP4244342A1 (en) * 2020-11-12 2023-09-20 Precision BioSciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene

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
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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