DK3116997T3 - Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10) - Google Patents

Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10) Download PDF

Info

Publication number
DK3116997T3
DK3116997T3 DK15714320.7T DK15714320T DK3116997T3 DK 3116997 T3 DK3116997 T3 DK 3116997T3 DK 15714320 T DK15714320 T DK 15714320T DK 3116997 T3 DK3116997 T3 DK 3116997T3
Authority
DK
Denmark
Prior art keywords
lca10
crispr
cas
treat
lever
Prior art date
Application number
DK15714320.7T
Other languages
English (en)
Inventor
Morgan L Maeder
David A Bumcrot
Shen Shen
Original Assignee
Editas Medicine Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Editas Medicine Inc filed Critical Editas Medicine Inc
Application granted granted Critical
Publication of DK3116997T3 publication Critical patent/DK3116997T3/da

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • C12N15/1024In vivo mutagenesis using high mutation rate "mutator" host strains by inserting genetic material, e.g. encoding an error prone polymerase, disrupting a gene for mismatch repair
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Coloring Foods And Improving Nutritive Qualities (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
DK15714320.7T 2014-03-10 2015-03-10 Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10) DK3116997T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461950733P 2014-03-10 2014-03-10
US201462036576P 2014-08-12 2014-08-12
PCT/US2015/019790 WO2015138510A1 (en) 2014-03-10 2015-03-10 Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)

Publications (1)

Publication Number Publication Date
DK3116997T3 true DK3116997T3 (da) 2019-08-19

Family

ID=52811198

Family Applications (1)

Application Number Title Priority Date Filing Date
DK15714320.7T DK3116997T3 (da) 2014-03-10 2015-03-10 Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)

Country Status (6)

Country Link
US (4) US9938521B2 (da)
EP (2) EP3553176A1 (da)
CA (1) CA2948728A1 (da)
DK (1) DK3116997T3 (da)
ES (1) ES2745769T3 (da)
WO (1) WO2015138510A1 (da)

Families Citing this family (177)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9585920B2 (en) 2011-02-04 2017-03-07 Katherine Rose Kovarik Method and system for treating cancer cachexia
US9457077B2 (en) 2009-11-18 2016-10-04 Katherine Rose Kovarik Method and system for targeting the microbiome to promote health and treat allergic and inflammatory diseases
US12257272B2 (en) 2015-12-24 2025-03-25 Seed Health, Inc. Method and system for reducing the likelihood of developing depression in an individual
US10086018B2 (en) 2011-02-04 2018-10-02 Joseph E. Kovarik Method and system for reducing the likelihood of colorectal cancer in a human being
US11844720B2 (en) 2011-02-04 2023-12-19 Seed Health, Inc. Method and system to reduce the likelihood of dental caries and halitosis
US11951140B2 (en) 2011-02-04 2024-04-09 Seed Health, Inc. Modulation of an individual's gut microbiome to address osteoporosis and bone disease
US10548761B2 (en) 2011-02-04 2020-02-04 Joseph E. Kovarik Method and system for reducing the likelihood of colorectal cancer in a human being
US11523934B2 (en) 2011-02-04 2022-12-13 Seed Health, Inc. Method and system to facilitate the growth of desired bacteria in a human's mouth
US11419903B2 (en) 2015-11-30 2022-08-23 Seed Health, Inc. Method and system for reducing the likelihood of osteoporosis
US11951139B2 (en) 2015-11-30 2024-04-09 Seed Health, Inc. Method and system for reducing the likelihood of osteoporosis
US9730967B2 (en) 2011-02-04 2017-08-15 Katherine Rose Kovarik Method and system for treating cancer cachexia
US9987224B2 (en) 2011-02-04 2018-06-05 Joseph E. Kovarik Method and system for preventing migraine headaches, cluster headaches and dizziness
US10512661B2 (en) 2011-02-04 2019-12-24 Joseph E. Kovarik Method and system for reducing the likelihood of developing liver cancer in an individual diagnosed with non-alcoholic fatty liver disease
US10314865B2 (en) 2011-02-04 2019-06-11 Katherine Rose Kovarik Method and system for treating cancer and other age-related diseases by extending the healthspan of a human
US10687975B2 (en) 2011-02-04 2020-06-23 Joseph E. Kovarik Method and system to facilitate the growth of desired bacteria in a human's mouth
US12533312B2 (en) 2011-02-04 2026-01-27 Seed Health, Inc. Method and system for preventing sore throat in humans
US12279989B2 (en) 2011-02-04 2025-04-22 Seed Health, Inc. Method and system for increasing beneficial bacteria and decreasing pathogenic bacteria in the oral cavity
US11998479B2 (en) 2011-02-04 2024-06-04 Seed Health, Inc. Method and system for addressing adverse effects on the oral microbiome and restoring gingival health caused by sodium lauryl sulphate exposure
US10842834B2 (en) 2016-01-06 2020-11-24 Joseph E. Kovarik Method and system for reducing the likelihood of developing liver cancer in an individual diagnosed with non-alcoholic fatty liver disease
US10245288B2 (en) 2011-02-04 2019-04-02 Joseph E. Kovarik Method and system for reducing the likelihood of developing NASH in an individual diagnosed with non-alcoholic fatty liver disease
US11273187B2 (en) 2015-11-30 2022-03-15 Joseph E. Kovarik Method and system for reducing the likelihood of developing depression in an individual
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
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
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US11969445B2 (en) 2013-12-20 2024-04-30 Seed Health, Inc. Probiotic composition and method for controlling excess weight, obesity, NAFLD and NASH
US11826388B2 (en) 2013-12-20 2023-11-28 Seed Health, Inc. Topical application of Lactobacillus crispatus to ameliorate barrier damage and inflammation
US12005085B2 (en) 2013-12-20 2024-06-11 Seed Health, Inc. Probiotic method and composition for maintaining a healthy vaginal microbiome
US11998574B2 (en) 2013-12-20 2024-06-04 Seed Health, Inc. Method and system for modulating an individual's skin microbiome
US11839632B2 (en) 2013-12-20 2023-12-12 Seed Health, Inc. Topical application of CRISPR-modified bacteria to treat acne vulgaris
US12246043B2 (en) 2013-12-20 2025-03-11 Seed Health, Inc. Topical application to treat acne vulgaris
US11980643B2 (en) 2013-12-20 2024-05-14 Seed Health, Inc. Method and system to modify an individual's gut-brain axis to provide neurocognitive protection
US11833177B2 (en) 2013-12-20 2023-12-05 Seed Health, Inc. Probiotic to enhance an individual's skin microbiome
US12329783B2 (en) 2013-12-20 2025-06-17 Seed Health, Inc. Method and system to improve the health of a person's skin microbiome
US11315659B2 (en) * 2014-01-27 2022-04-26 Georgia Tech Research Corporation Methods and systems for identifying nucleotide-guided nuclease off-target sites
WO2015113063A1 (en) * 2014-01-27 2015-07-30 Georgia Tech Research Corporation Methods and systems for identifying crispr/cas off-target sites
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
US11339437B2 (en) * 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
ES2745769T3 (es) 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
EP3122880B1 (en) 2014-03-26 2021-05-05 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
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
US10190106B2 (en) * 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
KR102888521B1 (ko) * 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
BR112017024514A2 (pt) * 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
WO2017004261A1 (en) * 2015-06-29 2017-01-05 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
JP6799586B2 (ja) 2015-08-28 2020-12-16 ザ ジェネラル ホスピタル コーポレイション 遺伝子操作CRISPR−Cas9ヌクレアーゼ
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
EP3159407A1 (en) * 2015-10-23 2017-04-26 Silence Therapeutics (London) Ltd Guide rnas, methods and uses
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
CA3005474A1 (en) 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
JP2019508051A (ja) 2016-03-14 2019-03-28 エディタス・メディシン、インコーポレイテッド β異常ヘモグロビン症を治療するためのCRISPR/CAS関連方法および組成物
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
US11597924B2 (en) 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
CN113831407B (zh) 2016-05-20 2024-06-11 瑞泽恩制药公司 用于使用多个引导rna来破坏免疫耐受性的方法
US12595478B2 (en) 2016-06-29 2026-04-07 The Broad Institute, Inc. Crispr-Cas systems having destabilization domain
KR20190039703A (ko) * 2016-07-05 2019-04-15 더 존스 홉킨스 유니버시티 Crispr/cas9-기반 조성물 및 망막 변성을 치료하기 위한 방법
CA3029860A1 (en) * 2016-07-05 2018-01-11 The Johns Hopkins University Compositions and methods comprising improvements of crispr guide rnas using the h1 promoter
US20180010117A1 (en) * 2016-07-07 2018-01-11 Sangamo Therapeutics, Inc. Targeted treatment of leber congenital amourosis
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
BR112019001887A2 (pt) * 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
CN110139654A (zh) * 2016-11-03 2019-08-16 优美佳生物技术有限公司 用于细胞重编程的方法和组合物
CN110168084A (zh) 2016-12-05 2019-08-23 爱迪塔斯医药公司 用于内源和来源DNA的单发指导RNA(ogRNA)靶向的系统和方法
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CA3048434A1 (en) 2016-12-30 2018-07-05 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN110709095B (zh) 2017-04-05 2024-05-03 马萨诸塞大学 小基因治疗
US11591589B2 (en) 2017-04-21 2023-02-28 The General Hospital Corporation Variants of Cpf1 (Cas12a) with altered PAM specificity
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
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
EP3630849A4 (en) 2017-05-25 2021-01-13 The General Hospital Corporation BIPARTITE BASIC EDITOR (BBE) AND TYPE II-C-CAS9 ZINC FINGER EDITOR ARCHITECTURES
CN109030429B (zh) * 2017-06-09 2022-06-21 南京大学 一种可对活细胞内microRNA21进行原位成像的纳米放大自参比探针
EP3652310B1 (en) * 2017-07-10 2023-05-03 The Hospital for Sick Children Gene editing system for correcting splicing defects
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
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
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
DK3701028T3 (da) 2017-10-24 2024-11-11 Editas Medicine Inc Systemer og fremgangsmåder til behandling af hyper-igm-syndrom
WO2019089884A2 (en) 2017-11-01 2019-05-09 Editas Medicine, Inc. Methods, compositions and components for crispr-cas9 editing of tgfbr2 in t cells for immunotherapy
EP3707258A1 (en) 2017-11-06 2020-09-16 Editas Medicine, Inc. Methods, compositions and components for crispr-cas9 editing of cblb in t cells for immunotherapy
US20200263206A1 (en) 2017-11-07 2020-08-20 Editas Medicine, Inc. Targeted integration systems and methods for the treatment of hemoglobinopathies
EP3724326A1 (en) 2017-12-11 2020-10-21 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
EP3746554A1 (en) 2018-01-30 2020-12-09 Editas Medicine, Inc. Systems and methods for modulating chromosomal rearrangements
WO2019152868A1 (en) 2018-02-01 2019-08-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus (hsv) related keratitis
EP3762496A2 (en) 2018-03-07 2021-01-13 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3093701A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR102907245B1 (ko) 2018-03-14 2026-01-05 에디타스 메디신, 인코포레이티드 혈색소병증 치료를 위한 시스템 및 방법
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
KR102866133B1 (ko) 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020027982A1 (en) 2018-08-02 2020-02-06 Editas Medicine, Inc. Compositions and methods for treating cep290-associated disease
US12497611B2 (en) 2018-08-17 2025-12-16 Yale University Compositions and methods for high-throughput activation screening to boost T cell effector function
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
SG11202103917VA (en) 2018-10-16 2021-05-28 Blueallele Llc Methods for targeted insertion of dna in genes
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
RU2712497C1 (ru) * 2018-11-26 2020-01-29 Автономная некоммерческая образовательная организация высшего образования Сколковский институт науки и технологий Средство разрезания ДНК на основе Cas9 белка из биотехнологически значимой бактерии Clostridium cellulolyticum
EP3887521A1 (en) 2018-11-29 2021-10-06 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN113544266A (zh) 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3918071A1 (en) 2019-02-01 2021-12-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions targeting virus genomes
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
PE20211959A1 (es) 2019-02-15 2021-09-30 Editas Medicine Inc Celulas asesinas naturales (nk) modificadas para inmunoterapia
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
PT3947684T (pt) 2019-03-29 2025-05-19 Ionis Pharmaceuticals Inc Compostos e métodos para modular ube3a‑ats
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
WO2020236815A1 (en) 2019-05-20 2020-11-26 University Of Massachusetts Minigene therapy
JP7689949B2 (ja) 2019-10-03 2025-06-09 アーティサン ディベロップメント ラブズ インコーポレイテッド 操作されたデュアルガイド核酸を有するcrisprシステム
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4069829A1 (en) 2019-12-08 2022-10-12 Editas Medicine, Inc. Modified cells and methods for the treatment of hemoglobinopathies
EP3901262A1 (en) 2020-04-20 2021-10-27 Universität Regensburg Compositions for use in treating autosomal dominant best1-related retinopathies
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CN115956120A (zh) * 2020-05-12 2023-04-11 马萨诸塞大学 用于crispr基因组编辑的修饰的指导rna
EP4232567A1 (en) 2020-10-26 2023-08-30 Shoreline Biosciences, Inc. Methods of inducing antibody-dependent cellular cytotoxicity (adcc) using modified natural killer (nk) cells
AU2022227650A1 (en) 2021-02-25 2023-10-12 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
WO2022214632A1 (en) 2021-04-07 2022-10-13 Neoplants Sas Compositions and methods for indoor air remediation
CA3220294A1 (en) 2021-05-14 2022-11-17 Genkore Inc. Composition and method for treatment of lca10 using rna-guided nuclease
EP4419672A2 (en) 2021-06-01 2024-08-28 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
EP4359541A4 (en) 2021-06-23 2025-04-30 Editas Medicine, Inc. Engineered cells for therapy
JP2024528967A (ja) 2021-08-02 2024-08-01 エディタス・メディシン、インコーポレイテッド ヘモグロビン異常症の治療のためのシステム及び方法
JP2024537775A (ja) 2021-09-30 2024-10-16 アコーオス インコーポレイテッド Kcnq4関連難聴を治療するための組成物及び方法
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
EP4486881A1 (en) 2022-03-01 2025-01-08 Celyntra Therapeutics SA Composition and methods for transgene insertion
EP4522758A4 (en) 2022-05-13 2026-05-06 Ascidian Therapeutics Inc Abca4 trans-splicing molecules
WO2024026478A1 (en) * 2022-07-29 2024-02-01 Beam Therapeutics Inc. Compositions and methods for treating a congenital eye disease
EP4619525A1 (en) * 2022-11-16 2025-09-24 Editas Medicine, Inc. Methods for treating cep290 associated disease
EP4630554A1 (en) 2022-12-05 2025-10-15 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US12383615B2 (en) 2023-03-23 2025-08-12 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods
WO2024196965A1 (en) 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2024249348A2 (en) 2023-05-26 2024-12-05 Editas Medicine, Inc. Crispr-related methods and compositions targeting fl1-1 expression
WO2024249342A1 (en) 2023-05-26 2024-12-05 Editas Medicine, Inc. Crispr-related methods and compositions targeting ptpn2 expression
KR20260018068A (ko) 2023-05-26 2026-02-06 에디타스 메디신, 인코포레이티드 Cd70 발현을 표적화하는 crispr 관련 방법 및 조성물
WO2024254566A2 (en) 2023-06-08 2024-12-12 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2025166325A1 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2026006542A2 (en) 2024-06-26 2026-01-02 Yale University Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome
WO2026064475A1 (en) 2024-09-18 2026-03-26 Editas Medicine, Inc. Crispr cas-related methods and compositions targeting ptpn2 expression
WO2026064739A1 (en) 2024-09-23 2026-03-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2026064736A1 (en) 2024-09-23 2026-03-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy

Family Cites Families (113)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
US5869624A (en) 1993-03-26 1999-02-09 Progenics Pharmaceuticals, Inc. HIV-1 vaccines, antibody compositions related thereto, and therapeutic and prophylactic uses thereof
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
US5725493A (en) 1994-12-12 1998-03-10 Avery; Robert Logan Intravitreal medicine delivery
US6299895B1 (en) 1997-03-24 2001-10-09 Neurotech S.A. Device and method for treating ophthalmic diseases
US6203986B1 (en) 1998-10-22 2001-03-20 Robert H. Singer Visualization of RNA in living cells
EP1154691A4 (en) 1999-01-05 2004-07-07 Massachusetts Eye & Ear Infirm TARGETED ADMINISTRATION OF REGULATED RELEASE MEDICINE TO RETINA AND CHOROID THROUGH SCLEROTIC
US6416777B1 (en) 1999-10-21 2002-07-09 Alcon Universal Ltd. Ophthalmic drug delivery device
CA2383499C (en) 1999-10-21 2009-11-24 Alcon Universal Ltd. Drug delivery device
KR100381902B1 (ko) 1999-11-29 2003-04-26 아이진 주식회사 녹내장-코딩 dna 및 단백질
EP1387671A1 (en) 2001-05-03 2004-02-11 MASSACHUSETTS EYE & EAR INFIRMARY Implantable drug delivery device and use thereof
WO2003072788A1 (en) 2002-02-21 2003-09-04 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
JP2006513694A (ja) 2002-06-11 2006-04-27 ザ スクリップス リサーチ インスティテュート 人工転写因子
DE602005024015D1 (de) 2004-08-23 2010-11-18 Alnylam Pharmaceuticals Inc Expressionskonstrukte mit mehreren rna-polymerase-iii-promotoren
WO2007014181A2 (en) 2005-07-25 2007-02-01 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
US9677123B2 (en) 2006-03-15 2017-06-13 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
CN101688241B (zh) 2007-03-02 2015-01-21 杜邦营养生物科学有限公司 具有改善的噬菌体抗性的培养物
AU2009231171A1 (en) 2008-04-02 2009-10-08 Fondazione Telethon Method of treating genetic disorders
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
US9115386B2 (en) 2008-09-26 2015-08-25 Children's Medical Center Corporation Selective oxidation of 5-methylcytosine by TET-family proteins
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
MX2012013037A (es) 2010-05-10 2013-07-29 Univ California Composiciones de endorribonucleasa y metodos de uso de las mismas.
GB201013153D0 (en) 2010-08-04 2010-09-22 Touchlight Genetics Ltd Primer for production of closed linear DNA
US9499592B2 (en) 2011-01-26 2016-11-22 President And Fellows Of Harvard College Transcription activator-like effectors
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
US9057148B2 (en) 2011-05-30 2015-06-16 Toyota Jidosha Kabushiki Kaisha High-strength polypropylene fiber and method for producing the same
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
WO2012168435A1 (en) * 2011-06-10 2012-12-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of leber congenital amaurosis
JP6214530B2 (ja) 2011-07-15 2017-10-18 ザ ジェネラル ホスピタル コーポレイション 転写活性化因子様エフェクターの組立て方法
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US20150166969A1 (en) 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
WO2013130824A1 (en) 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
WO2014022702A2 (en) 2012-08-03 2014-02-06 The Regents Of The University Of California Methods and compositions for controlling gene expression by rna processing
SG10201701601WA (en) 2012-08-29 2017-04-27 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
EP3789405A1 (en) 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
AU2013335451C1 (en) 2012-10-23 2024-07-04 Toolgen Incorporated Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
US20140186843A1 (en) 2012-12-12 2014-07-03 Massachusetts Institute Of Technology Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
EP2954042B1 (en) 2013-02-07 2017-12-06 The General Hospital Corporation Tale transcriptional activators
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
SG10201913068PA (en) 2013-06-04 2020-02-27 Harvard College Rna-guided transcriptional regulation
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
ES2929143T3 (es) 2013-07-09 2022-11-25 Harvard College Ingeniería genómica guiada por ARN múltiplex
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
EP3666892A1 (en) 2013-07-10 2020-06-17 President and Fellows of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
AU2014305218B2 (en) 2013-08-05 2020-01-16 Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum Recombinant AAV-Crumbs homologue composition and methods for treating LCA-8 and progressive RP
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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
EP3050366A4 (en) 2013-09-27 2017-05-31 Kyocera Corporation User equipment detection for energy saving cell activation
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
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
CA2930877A1 (en) 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用
CA2935032C (en) 2013-12-26 2024-01-23 The General Hospital Corporation Multiplex guide rnas
US11028388B2 (en) 2014-03-05 2021-06-08 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
US11339437B2 (en) * 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
ES2745769T3 (es) 2014-03-10 2020-03-03 Editas Medicine Inc Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10)
WO2015188056A1 (en) 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
ES2788426T3 (es) 2014-06-16 2020-10-21 Univ Johns Hopkins Composiciones y métodos para la expresión de ARNs de guía de CRISPR utilizando el promotor de H1
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
EP3274453B1 (en) 2015-03-26 2021-01-27 Editas Medicine, Inc. Crispr/cas-mediated gene conversion
US20160324987A1 (en) 2015-04-15 2016-11-10 Cedars-Sinai Medical Center Use of crispr/cas9 as in vivo gene therapy to generate targeted genomic disruptions in genes bearing dominant mutations for retinitis pigmentosa
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
BR112017024514A2 (pt) 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
WO2017040511A1 (en) 2015-08-31 2017-03-09 Agilent Technologies, Inc. Compounds and methods for crispr/cas-based genome editing by homologous recombination
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
KR20190039703A (ko) 2016-07-05 2019-04-15 더 존스 홉킨스 유니버시티 Crispr/cas9-기반 조성물 및 망막 변성을 치료하기 위한 방법
BR112019001887A2 (pt) * 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
CA3048434A1 (en) 2016-12-30 2018-07-05 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法

Also Published As

Publication number Publication date
US20150252358A1 (en) 2015-09-10
US11268086B2 (en) 2022-03-08
US12234449B2 (en) 2025-02-25
EP3116997B1 (en) 2019-05-15
US10253312B2 (en) 2019-04-09
US9938521B2 (en) 2018-04-10
US20230025279A1 (en) 2023-01-26
EP3116997A1 (en) 2017-01-18
WO2015138510A1 (en) 2015-09-17
US20200017849A1 (en) 2020-01-16
US20180195058A1 (en) 2018-07-12
ES2745769T3 (es) 2020-03-03
EP3553176A1 (en) 2019-10-16
CA2948728A1 (en) 2015-09-17
WO2015138510A8 (en) 2016-10-06

Similar Documents

Publication Publication Date Title
DK3116997T3 (da) Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
IL287952A (en) Compositions and methods for non-myeloablative conditioning
ZA202100808B (en) Methods and compositions for improving plant traits
IL251527A0 (en) Preparations of adenosine deaminase-2 (ada2), their variants and methods of using them
SG10202109655VA (en) Compositions and methods for immunooncology
EP3331582A4 (en) METHODS AND COMPOSITIONS FOR THE TREATMENT OF TRANSPLANTATION CELLS
DK3242957T3 (da) Bakterieholdige sammensætninger og fremgangsmåder til anvendelse af samme til behandling og/eller forebyggelse af gastrointestinale, metaboliske og/eller andre sygdomme
DK3134130T3 (da) Sammensætninger og fremgangsmåder til behandling af hæmoglobinopatier
DK3102673T3 (da) Fremgangsmåder og sammensætninger til behandling af beta-talassæmi
GB201513304D0 (en) Compositions and Methods
DK3172233T3 (da) Anti-cd74-antistoffer, sammensætninger omfattende anti-cd74- antistoffer og fremgangsmåder til anvendelse af anti-cd74-antistoffer
DK3371313T3 (da) Fremgangsmåder og sammensætninger til forbedret plantetransformation
DK4293009T3 (da) Fenfluraminsammensætninger og fremgangsmåder til fremstilling af samme
EP3253403A4 (en) Methods and compositions for improved cognition
DK3110446T3 (da) Fremgangsmåder og sammensætninger til behandling af Siglec-8-associerede sygdomme
IL259590A (en) Preparations and methods for manipulating plant growth
EP3139741A4 (en) Methods and compositions comprising 10-hydroxy-2-decenoic acid
EP3204426A4 (en) Compositions and methods for treating viral hemorrhagic fever
IL249640A0 (en) Preparations for the treatment of nosebleeds
PL3331359T3 (pl) Rolnicze kompozycje adiuwantowe i sposoby zastosowania takich kompozycji
GB2550632B (en) Compositions and uses and methods relating thereto
DK3386527T3 (da) Sammensætninger til anvendelse i behandling eller forbedring af neuroinflammation, neurodegeneration, neuropatisk smerte og migræne
DK3998067T3 (da) Sammensætninger og fremgangsmåder til behandling af homocystinuri
HK1255221A1 (zh) 使用卡多曲组合物进行治疗的方法
ZA201704942B (en) Compositions for improving budbreak and flowering