JP2017512767A5 - - Google Patents

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Publication number
JP2017512767A5
JP2017512767A5 JP2016556801A JP2016556801A JP2017512767A5 JP 2017512767 A5 JP2017512767 A5 JP 2017512767A5 JP 2016556801 A JP2016556801 A JP 2016556801A JP 2016556801 A JP2016556801 A JP 2016556801A JP 2017512767 A5 JP2017512767 A5 JP 2017512767A5
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JP
Japan
Prior art keywords
nuclease
medicament according
nucleases
seq
exon
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Pending
Application number
JP2016556801A
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English (en)
Japanese (ja)
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JP2017512767A (ja
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Publication date
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Priority claimed from PCT/US2015/020205 external-priority patent/WO2015138739A2/en
Publication of JP2017512767A publication Critical patent/JP2017512767A/ja
Publication of JP2017512767A5 publication Critical patent/JP2017512767A5/ja
Pending legal-status Critical Current

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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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019152614A Division 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

Publications (2)

Publication Number Publication Date
JP2017512767A JP2017512767A (ja) 2017-05-25
JP2017512767A5 true JP2017512767A5 (enExample) 2018-04-26

Family

ID=54072591

Family Applications (5)

Application Number Title Priority Date Filing Date
JP2016556801A Pending JP2017512767A (ja) 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
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

Family Applications After (4)

Application Number Title Priority Date Filing Date
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

Country Status (8)

Country Link
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)

Families Citing this family (55)

* 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
DK2986729T3 (en) 2013-04-16 2018-10-29 Regeneron Pharma TARGETED MODIFICATION OF ROOT THROUGH
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
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
WO2015088643A1 (en) 2013-12-11 2015-06-18 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CA2968440A1 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
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
US10603363B2 (en) * 2015-09-08 2020-03-31 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
EP4108255A1 (en) 2015-10-05 2022-12-28 Precision Biosciences, Inc. Genetically-modified cells comprising a modified human t cell receptor alpha constant region gene
ES2883116T3 (es) * 2015-10-05 2021-12-07 Prec Biosciences Inc Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
US20170332610A1 (en) 2016-05-20 2017-11-23 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 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
CN110382695A (zh) * 2016-11-28 2019-10-25 得克萨斯州大学系统董事会 通过crispr/cpf1介导的基因编辑来预防肌营养不良
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
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising 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
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
JP2020529834A (ja) 2017-06-30 2020-10-15 プレシジョン バイオサイエンシズ，インク． Ｔ細胞受容体アルファ遺伝子の改変されたイントロンを含む遺伝子改変ｔ細胞
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
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
JP7304888B2 (ja) 2018-04-12 2023-07-07 プレシジョン バイオサイエンシズ，インク． ヒトｔ細胞受容体アルファ定常領域遺伝子に特異性を有する最適化された操作されたヌクレアーゼ
US12157760B2 (en) 2018-05-23 2024-12-03 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
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020106633A1 (en) * 2018-11-19 2020-05-28 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
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20220005555A (ko) 2019-05-07 2022-01-13 프리시젼 바이오사이언시스 인코포레이티드 인식 서열에 대한 조작된 메가뉴클레아제의 최적화
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20210361778A1 (en) * 2020-04-06 2021-11-25 Homology Medicines, Inc. Adeno-associated virus compositions 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
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
WO2021231259A1 (en) 2020-05-11 2021-11-18 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
IL302733B2 (en) * 2020-11-12 2025-05-01 Prec Biosciences Inc Engineered nucleases with specificity for recognition sequences in the dystrophin gene
EP4444886A1 (en) * 2021-12-08 2024-10-16 Mammoth Biosciences, Inc. Systems and uses thereof for the treatment of dmd-associated diseases
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

Family Cites Families (20)

* 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.
EP2650366B1 (en) 2005-10-18 2017-03-22 Precision Biosciences Rationally designed meganucleases with altered sequence specificity and DNA-binding affinity
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)
DK2313498T3 (en) 2008-07-14 2017-05-22 Prec Biosciences Inc RECOGNITION SEQUENCES FOR I-CREI-DERIVED MECHANUCLEASES AND APPLICATIONS THEREOF
WO2011036640A2 (en) * 2009-09-24 2011-03-31 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
EP2569424A1 (en) * 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
EP2633040B1 (en) * 2010-10-27 2019-07-10 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
ES2713529T3 (es) * 2011-06-01 2019-05-22 Prec Biosciences Inc Procedimientos y productos para la producción de líneas celulares de mamífero modificadas con transgenes amplificados
DK2766483T3 (da) * 2011-10-10 2022-04-19 Hospital For Sick Children Fremgangsmåder og sammensætninger til screening for og behandling af udviklingsforstyrrelser
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
WO2013163628A2 (en) * 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
EP2684892A1 (en) * 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
EP2914728B1 (en) * 2012-11-01 2020-07-08 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
ES3029138T3 (en) * 2013-06-05 2025-06-23 Univ Duke Rna-guided gene editing and gene regulation
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
IL302733B2 (en) * 2020-11-12 2025-05-01 Prec Biosciences Inc Engineered nucleases with specificity for recognition sequences in the dystrophin gene

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