JP2017512767A5 - - Google Patents

Download PDF

Info

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
Authority
JP
Japan
Prior art keywords
nuclease
medicament according
nucleases
seq
exon
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2016556801A
Other languages
English (en)
Japanese (ja)
Other versions
JP2017512767A (ja
Filing date
Publication date
Application filed filed Critical
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

Links

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
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
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
CN111218447B (zh) 2013-11-07 2024-10-11 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
MX388127B (es) 2013-12-11 2025-03-19 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
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
EP3221457B1 (en) 2014-11-21 2019-03-20 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
CA2981508A1 (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
CA2997909A1 (en) * 2015-09-08 2017-03-16 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
ES2803728T3 (es) 2015-10-05 2021-01-29 Prec Biosciences Inc Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado
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
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
SG10202102885UA (en) 2016-05-20 2021-05-28 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
AU2017308889B2 (en) 2016-08-09 2023-11-09 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
KR20240007715A (ko) 2016-10-14 2024-01-16 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
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
EP4775669A2 (en) 2017-06-30 2026-07-15 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
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
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
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
WO2020191241A1 (en) 2019-03-19 2020-09-24 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
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
WO2021207077A1 (en) * 2020-04-06 2021-10-14 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
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
WO2021231259A1 (en) 2020-05-11 2021-11-18 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
KR20250099412A (ko) 2020-11-12 2025-07-01 프리시젼 바이오사이언시스 인코포레이티드 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제
EP4444886A4 (en) * 2021-12-08 2026-08-12 Mammoth Biosciences Inc Systems and their uses for the treatment of diseases associated with DMD
TW202600818A (zh) * 2024-02-26 2026-01-01 日商日冷食品股份有限公司 包含經修飾dna的細胞之製作方法、包含該細胞的生物體之製作方法,及基因產物之生產方法
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

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060206949A1 (en) * 2003-01-28 2006-09-14 Sylvain Arnould Custom-made meganuclease and use thereof
DK3246403T3 (da) 2005-10-18 2020-10-26 Prec Biosciences Rationelt designede meganucleaser med ændret sekvens-specificitet og dna-bindende affinitet
ES2422291T3 (es) 2007-10-31 2013-09-10 Prec Biosciences Inc Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
JP2011527906A (ja) 2008-07-14 2011-11-10 プレシジョン バイオサイエンシズ,インク. I−CreI由来メガヌクレアーゼの認識配列およびその使用
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
SG189482A1 (en) * 2010-10-27 2013-05-31 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
EP3683319A1 (en) * 2011-06-01 2020-07-22 Precision Biosciences, Inc. Methods and products for producing engineered mammalian cell lines with amplified transgenes
WO2013054200A2 (en) * 2011-10-10 2013-04-18 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
EP2841572B1 (en) * 2012-04-27 2019-06-19 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
CA2890110C (en) * 2012-11-01 2023-05-02 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
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University 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
EP4279084B1 (en) * 2015-10-28 2025-06-11 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

Similar Documents

Publication Publication Date Title
JP2019522461A5 (enExample)
WO2016094845A3 (en) Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
JP2017505117A5 (enExample)
JP2016501532A5 (enExample)
WO2015138739A3 (en) Dystrophin gene exon deletion using engineered nucleases
RU2016123086A (ru) Способы и композиции для лечения болезни хантингтона
Persico et al. Autism genetics
JP2016538885A5 (enExample)
JP2019524149A5 (enExample)
JP2020518276A5 (enExample)
JP2018143253A5 (enExample)
JP2016116520A5 (enExample)
WO2018192982A3 (en) METHODS OF GENERATING ADENO-ASSOCIATED VIRAL VECTORS
JP2018534950A5 (enExample)
JP2016500262A5 (enExample)
WO2016115543A3 (en) Novel micro-dystrophins and related methods of use
WO2015138357A3 (en) Compositions useful in treatment of otc deficency
JP2010523099A5 (enExample)
JP2016537341A5 (enExample)
WO2017053431A3 (en) Allele selective gene editing and uses thereof
JP2016521995A5 (enExample)
JP2016525888A5 (enExample)
FI3690056T3 (fi) Menetelmiä ja tuotteita nukleiinihapon valmistamiseksi ja viemiseksi
RU2017115838A (ru) Направляемое РНК уничтожение вируса JC человека и других полиомавирусов
JP2012136542A5 (enExample)