RU2759335C2 - Генное редактирование глубоких интронных мутаций - Google Patents

Генное редактирование глубоких интронных мутаций Download PDF

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RU2759335C2
RU2759335C2 RU2017143997A RU2017143997A RU2759335C2 RU 2759335 C2 RU2759335 C2 RU 2759335C2 RU 2017143997 A RU2017143997 A RU 2017143997A RU 2017143997 A RU2017143997 A RU 2017143997A RU 2759335 C2 RU2759335 C2 RU 2759335C2
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guide rna
cas
promoter
seq
mutation
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RU2017143997A
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Russian (ru)
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RU2017143997A (ru
RU2017143997A3 (enExample
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Госян ЖУАНЬ
Абрахам СКАРИА
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Джензим Корпорейшн
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RU2017143997A 2015-05-16 2016-04-15 Генное редактирование глубоких интронных мутаций RU2759335C2 (ru)

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US201562162720P 2015-05-16 2015-05-16
US62/162,720 2015-05-16
PCT/US2016/027987 WO2016186772A2 (en) 2015-05-16 2016-04-15 Gene editing of deep intronic mutations

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RU2759335C2 true RU2759335C2 (ru) 2021-11-12

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US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
DK3116997T3 (da) 2014-03-10 2019-08-19 Editas Medicine Inc Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
EP3540061A1 (en) 2014-04-02 2019-09-18 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
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.
KR102535217B1 (ko) 2015-04-24 2023-05-19 에디타스 메디신, 인코포레이티드 Ｃａｓ9 분자/가이드 ｒｎａ 분자 복합체의 평가
WO2016186772A2 (en) * 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
EP4115894A1 (en) * 2015-06-19 2023-01-11 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
IL262888B1 (en) 2016-05-20 2025-10-01 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide RNAs
CA3032822A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
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
IT201600102542A1 (it) * 2016-10-12 2018-04-12 Univ Degli Studi Di Trento Plasmide e sistema lentivirale contenente un circuito autolimitante della Cas9 che ne incrementa la sicurezza.
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
CA3046076A1 (en) 2016-12-05 2018-06-14 Editas Medicine, Inc. Systems and methods for one-shot guide rna (ogrna) targeting of endogenous and source dna
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
EP3592853A1 (en) 2017-03-09 2020-01-15 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
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
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
EP3622073A4 (en) * 2017-05-09 2021-01-06 University of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
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
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
EP3684937A4 (en) 2017-09-22 2021-06-02 University of Massachusetts NEW DUAL SOD1 EXPRESSION VECTORS AND RELATED USES
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
JP7522656B2 (ja) 2017-11-21 2024-07-25 クリスパー セラピューティクス アーゲー 常染色体優性網膜色素変性の処置のための材料および方法
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3765614A1 (en) 2018-03-14 2021-01-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US20210017509A1 (en) * 2018-03-23 2021-01-21 The Trustees Of Columbia University In The City Of New York Gene Editing for Autosomal Dominant Diseases
CA3094828A1 (en) * 2018-03-23 2019-09-26 Massachusetts Eye And Ear Infirmary Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome
US12121563B2 (en) 2018-04-09 2024-10-22 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3833680A4 (en) * 2018-05-30 2022-05-25 The Regents of the University of California GENE EDIT OF MONOGENIC DISEASES IN HUMAN HEMATOPOIETIC STEM CELLS - CORRECTION OF X-LINKED HYPER-IGM SYNDROME (XHIM)
GB201809588D0 (en) * 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
CA3108376A1 (en) * 2018-08-02 2020-02-06 Editas Medicine, Inc. Compositions and methods for treating cep290-associated disease
KR102158428B1 (ko) * 2018-08-16 2020-09-22 (주)라트바이오 인위적 뉴클레아제를 생산하는 형질전환 배아 및 형질전환 동물의 제작 방법
US12472268B2 (en) 2018-10-01 2025-11-18 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
JP7695192B2 (ja) 2018-10-08 2025-06-18 アレン インスティテュート 介在ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
JP7704998B2 (ja) * 2018-11-21 2025-07-09 武田薬品工業株式会社 組換えウイルスベクター及びそれの産生のための核酸
IT201800020230A1 (it) * 2018-12-19 2020-06-19 Univ Degli Studi Di Siena Sistema CRISPR-Cas per la terapia genica.
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020176747A1 (en) * 2019-02-28 2020-09-03 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
KR20220017887A (ko) * 2019-03-27 2022-02-14 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Cas9/rnp 및 aav 바이러스의 조합을 사용한 암 면역요법을 위한 키메라 항원 수용체 (car)-일차 nk 세포의 생성
KR20210113393A (ko) * 2019-03-28 2021-09-15 주식회사 툴젠 혈액응고인자 viii 유전자 역위 보정에 의한 혈우병 치료용 조성물
KR20220009950A (ko) * 2019-04-15 2022-01-25 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 효소 발현의 조절 및 자가-불활성화를 위한 조성물 그리고 효소의 오프-타깃 활동성의 조정 방법
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
CN111370055B (zh) * 2020-03-05 2023-05-23 中南大学 内含子保留预测模型建立方法及其预测方法
WO2021195218A1 (en) * 2020-03-24 2021-09-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
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
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
EP4301462A1 (en) * 2021-03-04 2024-01-10 Research Institute at Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
US20220325298A1 (en) * 2021-04-12 2022-10-13 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Crispr epigenetic therapeutics for pain management
JP7780172B2 (ja) * 2021-07-30 2025-12-04 国立研究開発法人理化学研究所 筋萎縮性側索硬化症におけるｃｅｐ２９０遺伝子の変異
CN114306277B (zh) * 2021-11-02 2023-08-04 中国人民解放军空军军医大学 一种靶向psma的巨噬细胞膜包裹的仿生纳米药物载体及应用
EP4223877A1 (en) 2022-02-08 2023-08-09 Eberhard Karls Universität Tübingen Medizinische Fakultät System and method for editing genomic dna to modulate splicing
WO2025135916A1 (ko) * 2023-12-22 2025-06-26 주식회사 툴젠 낮은 면역원성을 가지는 cas9 단백질 변이체

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015070083A1 (en) * 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP2369002A1 (en) 1999-08-09 2011-09-28 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
NZ561656A (en) 2002-05-01 2009-03-31 Univ Florida Improved rAAV expression systems for genetic modification of specific capsid proteins
PT1625210E (pt) 2003-05-21 2011-03-15 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US7407801B2 (en) * 2003-12-05 2008-08-05 University Of Iowa Research Foundation Truncated CMV promoters and vectors containing same
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
CA2720178A1 (en) 2008-04-02 2009-10-08 Fondazione Telethon Method of treating genetic disorders
HUE054940T2 (hu) 2009-06-16 2021-10-28 Genzyme Corp Javított eljárások rekombináns AAV-vektorok tisztítására
WO2012099540A1 (en) * 2011-01-17 2012-07-26 Agency For Science, Technology And Research Cmv promoter variants
SG10201601110VA (en) 2011-02-17 2016-03-30 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
AU2014281026B2 (en) * 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
RU2716421C2 (ru) * 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка, применение и применения в терапии систем crispr-cas и композиций для целенаправленного воздействия на нарушения и заболевания с использованием вирусных компонентов
AU2014362245A1 (en) * 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
DK3116997T3 (da) * 2014-03-10 2019-08-19 Editas Medicine Inc Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
WO2016186772A2 (en) * 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
EP4115894A1 (en) * 2015-06-19 2023-01-11 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
US11530421B2 (en) * 2016-02-01 2022-12-20 The Regents Of The University Of California Self-inactivating endonuclease-encoding nucleic acids and methods of using the same

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015070083A1 (en) * 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ANN RAN F et al., Genome engineering using the CRISPR-Cas9 system, NATURE PROTOCOLS, NATURE PUBLISHING GROUP, 2013, vol. 8, no. 11, pp. 2281-2308. *
MAEDER M.L. et al., 687. Therapeutic correction of an LCA-causing splice defect in the CEP290 gene by CRISPR/Cas-mediated genome editing, Molecular Therapy, May 13, 2015, Supplement 1, 23: S273-S274. *
OUSTEROUT D.G. et al., Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy, Nature communications, 2015, 6(1): 1-13. *
OUSTEROUT D.G. et al., Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy, Nature communications, 2015, 6(1): 1-13. ANN RAN F et al., Genome engineering using the CRISPR-Cas9 system, NATURE PROTOCOLS, NATURE PUBLISHING GROUP, 2013, vol. 8, no. 11, pp. 2281-2308. ПУГАЧ К.С. и др., CRISPR-системы адаптивного иммунитета прокариот, Молекулярная биология, март-апрель 2012, 46(2):195-203. *
ПУГАЧ К.С. и др., CRISPR-системы адаптивного иммунитета прокариот, Молекулярная биология, март-апрель 2012, 46(2):195-203. *

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