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 PDFInfo
- 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
Links
- 201000004569 Blindness Diseases 0.000 title 1
- 108091033409 CRISPR Proteins 0.000 title 1
- 238000010354 CRISPR gene editing Methods 0.000 title 1
- 206010010356 Congenital anomaly Diseases 0.000 title 1
- 206010001902 amaurosis Diseases 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
- C12N15/1024—In 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods 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)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (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)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Coloring Foods And Improving Nutritive Qualities (AREA)
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 (165)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US9585920B2 (en) | 2011-02-04 | 2017-03-07 | Katherine Rose Kovarik | Method and system for treating cancer cachexia |
| 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 |
| 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 |
| US11844720B2 (en) | 2011-02-04 | 2023-12-19 | Seed Health, Inc. | Method and system to reduce the likelihood of dental caries and halitosis |
| 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 |
| 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 |
| US9730967B2 (en) | 2011-02-04 | 2017-08-15 | Katherine Rose Kovarik | Method and system for treating cancer cachexia |
| US11951139B2 (en) | 2015-11-30 | 2024-04-09 | Seed Health, Inc. | Method and system for reducing the likelihood of osteoporosis |
| 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 |
| US12257272B2 (en) | 2015-12-24 | 2025-03-25 | Seed Health, Inc. | Method and system for reducing the likelihood of developing depression in an individual |
| 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 |
| 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 |
| US11951140B2 (en) | 2011-02-04 | 2024-04-09 | Seed Health, Inc. | Modulation of an individual's gut microbiome to address osteoporosis and bone disease |
| US9987224B2 (en) | 2011-02-04 | 2018-06-05 | Joseph E. Kovarik | Method and system for preventing migraine headaches, cluster headaches and dizziness |
| 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 |
| 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 |
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| 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 |
| EP2971041B1 (en) | 2013-03-15 | 2018-11-28 | 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 |
| SI3456831T1 (sl) | 2013-04-16 | 2021-11-30 | Regeneron Pharmaceuticals, Inc., | Ciljna modifikacija podganjega genoma |
| US10011850B2 (en) | 2013-06-21 | 2018-07-03 | The General Hospital Corporation | Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing |
| 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 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| KR102523466B1 (ko) | 2013-11-07 | 2023-04-20 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| US9546384B2 (en) | 2013-12-11 | 2017-01-17 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a mouse genome |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| 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 |
| US12329783B2 (en) | 2013-12-20 | 2025-06-17 | Seed Health, Inc. | Method and system to improve the health of a person's skin microbiome |
| US11969445B2 (en) | 2013-12-20 | 2024-04-30 | Seed Health, Inc. | Probiotic composition and method for controlling excess weight, obesity, NAFLD and NASH |
| 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 |
| US11998574B2 (en) | 2013-12-20 | 2024-06-04 | Seed Health, Inc. | Method and system for modulating an individual's skin microbiome |
| US11833177B2 (en) | 2013-12-20 | 2023-12-05 | Seed Health, Inc. | Probiotic to enhance an individual's skin microbiome |
| 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 |
| US10354746B2 (en) * | 2014-01-27 | 2019-07-16 | Georgia Tech Research Corporation | Methods and systems for identifying CRISPR/Cas off-target sites |
| US11315659B2 (en) * | 2014-01-27 | 2022-04-26 | Georgia Tech Research Corporation | Methods and systems for identifying nucleotide-guided nuclease off-target sites |
| 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 |
| US9938521B2 (en) | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| 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 |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| CN107208078B (zh) | 2014-11-21 | 2021-07-16 | 瑞泽恩制药公司 | 使用成对向导rna进行靶向遗传修饰的方法和组合物 |
| EP3985115A1 (en) | 2014-12-12 | 2022-04-20 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
| 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 |
| AU2015369725A1 (en) | 2014-12-24 | 2017-06-29 | Massachusetts Institute Of Technology | CRISPR having or associated with destabilization domains |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| JP6873911B2 (ja) * | 2015-04-06 | 2021-05-19 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 初代細胞において標的核酸の遺伝子調節を誘導するためにインビトロで行う方法 |
| CN114657180A (zh) * | 2015-05-16 | 2022-06-24 | 建新公司 | 深内含子突变的基因编辑 |
| CA3012607A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Crispr enzymes and systems |
| AU2016285724A1 (en) * | 2015-06-29 | 2017-11-16 | Ionis Pharmaceuticals, Inc. | Modified CRISPR RNA and modified single CRISPR RNA and uses thereof |
| HK1257676A1 (zh) | 2015-08-28 | 2019-10-25 | The General Hospital Corporation | 工程化 crispr-cas 9 核酸酶 |
| US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| US9926546B2 (en) | 2015-08-28 | 2018-03-27 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| CA3002827A1 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| EP3159407A1 (en) * | 2015-10-23 | 2017-04-26 | Silence Therapeutics (London) Ltd | Guide rnas, methods and uses |
| AU2016355343C1 (en) | 2015-11-19 | 2024-10-03 | Lloyd G. Mitchell | Compositions and methods for correction of heritable ocular disease |
| EA201891317A3 (ru) | 2015-11-30 | 2019-04-30 | Дьюк Юниверсити | Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения |
| CN117821458A (zh) | 2016-03-14 | 2024-04-05 | 爱迪塔斯医药公司 | 用于治疗β-血红蛋白病的CRISPR/CAS相关方法和组合物 |
| EP3219799A1 (en) | 2016-03-17 | 2017-09-20 | IMBA-Institut für Molekulare Biotechnologie GmbH | Conditional crispr sgrna expression |
| WO2017165826A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
| WO2017165862A1 (en) * | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| EP3443081A4 (en) | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE |
| EP3457840B1 (en) | 2016-05-20 | 2024-04-10 | Regeneron Pharmaceuticals, Inc. | Methods for breaking immunological tolerance using multiple guide rnas |
| AU2017292667A1 (en) * | 2016-07-05 | 2019-02-21 | The Johns Hopkins University | Compositions and methods comprising improvements of CRISPR guide RNAs using the H1 promoter |
| AU2017293773A1 (en) * | 2016-07-05 | 2019-02-21 | The Johns Hopkins University | CRISPR/Cas9-based compositions and methods for treating retinal degenerations |
| US20180010117A1 (en) * | 2016-07-07 | 2018-01-11 | Sangamo Therapeutics, Inc. | Targeted treatment of leber congenital amourosis |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| CA3032822A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| KR102827276B1 (ko) | 2016-08-03 | 2025-07-01 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| JP7201153B2 (ja) | 2016-08-09 | 2023-01-10 | プレジデント アンド フェローズ オブ ハーバード カレッジ | プログラム可能cas9-リコンビナーゼ融合タンパク質およびその使用 |
| CN110114461A (zh) | 2016-08-17 | 2019-08-09 | 博德研究所 | 新型crispr酶和系统 |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | 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 |
| BR112019009116A2 (pt) * | 2016-11-03 | 2019-10-15 | Univ California | métodos e composições para reprogramação celular |
| 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 |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| WO2018126176A1 (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 |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | 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 |
| WO2018165629A1 (en) | 2017-03-10 | 2018-09-13 | 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 |
| BR112019019655A2 (pt) | 2017-03-23 | 2020-04-22 | Harvard College | editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico |
| JP7307480B2 (ja) | 2017-04-05 | 2023-07-12 | ユニバーシティ オブ マサチューセッツ | ミニ遺伝子療法 |
| EP4481049A3 (en) | 2017-04-21 | 2025-03-19 | The General Hospital Corporation | Variants of cpf1 (cas12a) with altered pam specificity |
| EP3615672A1 (en) | 2017-04-28 | 2020-03-04 | Editas Medicine, Inc. | Methods and systems for analyzing guide rna molecules |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | 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 |
| JP2020521446A (ja) | 2017-05-25 | 2020-07-27 | ザ ジェネラル ホスピタル コーポレイション | 二部塩基エディター(bbe)構造およびii型−c−cas9ジンクフィンガー編集 |
| 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 |
| US20210388332A1 (en) * | 2017-07-10 | 2021-12-16 | The Hospital For Sick Children | Gene editing system for correcting splicing defects |
| WO2019014564A1 (en) | 2017-07-14 | 2019-01-17 | Editas Medicine, Inc. | SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES |
| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物 |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| CN111757937A (zh) | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| JP2021500080A (ja) | 2017-10-24 | 2021-01-07 | エディタス メディシン,インコーポレーテッド | 高IgM症候群を処置するためのシステム及び方法 |
| AU2018358250B2 (en) | 2017-11-01 | 2025-06-05 | Editas Medicine, Inc. | Methods, compositions and components for CRISPR-CAS9 editing of TGFBR2 in T cells for immunotherapy |
| US20230137729A1 (en) | 2017-11-06 | 2023-05-04 | Editas Medicine, Inc. | Methods, compositions and components for crispr-cas9 editing of cblb in t cells for immunotherapy |
| EP3707266A1 (en) | 2017-11-07 | 2020-09-16 | 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 |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| WO2019152519A1 (en) | 2018-01-30 | 2019-08-08 | Editas Medicine, Inc. | Systems and methods for modulating chromosomal rearrangements |
| EP3746556A1 (en) | 2018-02-01 | 2020-12-09 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus (hsv) related keratitis |
| WO2019173654A2 (en) | 2018-03-07 | 2019-09-12 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| WO2019178416A1 (en) | 2018-03-14 | 2019-09-19 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| MX2020009487A (es) | 2018-03-14 | 2020-12-10 | Editas Medicine Inc | Sistemas y metodos para el tratamiento de hemoglobinopatias. |
| US10968257B2 (en) | 2018-04-03 | 2021-04-06 | The Broad Institute, Inc. | Target recognition motifs and uses thereof |
| BR112020021228A2 (pt) | 2018-04-17 | 2021-03-02 | The Trustees Of The University Of Pennsylvania | moléculas de união trans |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12338436B2 (en) | 2018-06-29 | 2025-06-24 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| WO2020027982A1 (en) | 2018-08-02 | 2020-02-06 | Editas Medicine, Inc. | Compositions and methods for treating cep290-associated disease |
| WO2020037142A1 (en) * | 2018-08-17 | 2020-02-20 | 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 |
| CA3114699A1 (en) | 2018-10-09 | 2020-04-16 | The University Of British Columbia | Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto |
| AU2019360182B2 (en) | 2018-10-16 | 2023-02-16 | Blueallele Corporation | Methods for targeted insertion of DNA in genes |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | 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 |
| EP3898958A1 (en) | 2018-12-17 | 2021-10-27 | The Broad Institute, Inc. | Crispr-associated transposase systems and methods of use thereof |
| 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 |
| WO2020163396A1 (en) | 2019-02-04 | 2020-08-13 | 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 |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| CN117431244A (zh) | 2019-03-29 | 2024-01-23 | Ionis制药公司 | 用于调节ube3a-ats的化合物和方法 |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | 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 |
| CA3153197A1 (en) | 2019-10-03 | 2021-04-08 | Ryan T. Gill | Crispr systems with engineered dual guide nucleic acids |
| WO2021072328A1 (en) | 2019-10-10 | 2021-04-15 | The Broad Institute, Inc. | Methods and compositions for prime editing rna |
| IL293643A (en) | 2019-12-08 | 2022-08-01 | 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 |
| EP4146804A1 (en) | 2020-05-08 | 2023-03-15 | The Broad Institute Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| AU2021271004A1 (en) * | 2020-05-12 | 2023-01-19 | University Of Massachusetts | Modified guide RNAs for CRISPR genome editing |
| WO2022093749A1 (en) | 2020-10-26 | 2022-05-05 | Editas Medicine, Inc. | Methods of inducing antibody-dependent cellular cytotoxicity (adcc) using modified natural killer (nk) cells |
| WO2022182801A1 (en) | 2021-02-25 | 2022-09-01 | Artisan Development Labs, Inc. | 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 |
| US20250179481A1 (en) | 2021-06-01 | 2025-06-05 | Celyntra Therapeutics Sa | Compositions and methods for targeting, editing, or modifying genes |
| WO2023014727A1 (en) | 2021-08-02 | 2023-02-09 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| KR20240067112A (ko) | 2021-09-30 | 2024-05-16 | 아카우오스, 인크. | Kcnq4-연관 청력손실을 치료하기 위한 조성물 및 방법 |
| US20230279442A1 (en) | 2021-12-15 | 2023-09-07 | Versitech Limited | Engineered cas9-nucleases and method of use thereof |
| WO2023167882A1 (en) | 2022-03-01 | 2023-09-07 | Artisan Development Labs, Inc. | Composition and methods for transgene insertion |
| IL316944A (en) | 2022-05-13 | 2025-01-01 | Ascidian Therapeutics Inc | ABCA4 cross-linking 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 |
| WO2024123842A1 (en) | 2022-12-05 | 2024-06-13 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| WO2024196965A1 (en) | 2023-03-23 | 2024-09-26 | Carbon Biosciences, Inc. | Parvovirus compositions and related methods for gene therapy |
| US12383615B2 (en) | 2023-03-23 | 2025-08-12 | Carbon Biosciences, Inc. | Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods |
| WO2024249346A1 (en) | 2023-05-26 | 2024-12-05 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting cd70 expression |
| WO2024249342A1 (en) | 2023-05-26 | 2024-12-05 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting ptpn2 expression |
| WO2024249348A2 (en) | 2023-05-26 | 2024-12-05 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting fl1-1 expression |
| 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 |
Family Cites Families (113)
| 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 |
| JP2002534139A (ja) | 1999-01-05 | 2002-10-15 | マサチューセッツ・アイ・アンド・イア・インファーマリー | 網膜と脈絡膜への経強膜徐放性薬剤標的送達 |
| ATE283013T1 (de) | 1999-10-21 | 2004-12-15 | Alcon Inc | Medikamentenzuführvorrichtung |
| US6416777B1 (en) | 1999-10-21 | 2002-07-09 | Alcon Universal Ltd. | Ophthalmic 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 |
| AU2003219847A1 (en) | 2002-02-21 | 2003-09-09 | The Wistar Institute Of Anatomy And Biology | Methods and compositions for reversibly controlling expression of target genes in cells |
| WO2003104414A2 (en) | 2002-06-11 | 2003-12-18 | The Scripps Research Institute | Artificial transcription factors |
| EP2270161A3 (en) | 2004-08-23 | 2011-02-02 | Alnylam Pharmaceuticals, Inc. | Multiple RNA polymerase III promoter expression constructs |
| 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 |
| NZ592231A (en) | 2007-03-02 | 2012-07-27 | Danisco | Methods to generate bacteriophage resistant bacterial strains and produce bacteriophage CRISPR loci phage mutants |
| AU2009231171A1 (en) | 2008-04-02 | 2009-10-08 | Fondazione Telethon | Method of treating genetic disorders |
| US8546553B2 (en) | 2008-07-25 | 2013-10-01 | 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 |
| WO2010037001A2 (en) | 2008-09-26 | 2010-04-01 | Immune Disease Institute, Inc. | 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 |
| 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 |
| CN103038338B (zh) | 2010-05-10 | 2017-03-08 | 加利福尼亚大学董事会 | 核糖核酸内切酶组合物及其使用方法 |
| AU2011256838B2 (en) | 2010-05-17 | 2014-10-09 | Sangamo Therapeutics, Inc. | Novel DNA-binding proteins and uses thereof |
| 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 |
| PL3254703T3 (pl) * | 2011-04-22 | 2020-10-05 | The Regents Of The University Of California | Wiriony wirusa towarzyszącego adenowirusom z różnymi kapsydami i sposoby ich zastosowania |
| EP2716799A4 (en) | 2011-05-30 | 2014-10-29 | Toyota Motor Co Ltd | HIGH-STRENGTH POLYPROPYLENE FIBER AND METHOD OF MANUFACTURING THEREOF |
| US20140113376A1 (en) | 2011-06-01 | 2014-04-24 | Rotem Sorek | Compositions and methods for downregulating prokaryotic genes |
| EP3378938A1 (en) | 2011-06-10 | 2018-09-26 | Institut National de la Sante et de la Recherche Medicale (INSERM) | 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 |
| NZ630900A (en) | 2012-02-24 | 2016-10-28 | Hutchinson Fred Cancer Res | Compositions and methods for the treatment of hemoglobinopathies |
| AU2013225950B2 (en) | 2012-02-29 | 2018-02-15 | Sangamo Therapeutics, 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 |
| EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | 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 |
| US20160046961A1 (en) | 2012-05-25 | 2016-02-18 | 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 |
| JP2015527889A (ja) | 2012-07-25 | 2015-09-24 | ザ ブロード インスティテュート, インコーポレイテッド | 誘導可能なdna結合タンパク質およびゲノム撹乱ツール、ならびにそれらの適用 |
| HK1207111A1 (en) | 2012-08-03 | 2016-01-22 | 加利福尼亚大学董事会 | Methods and compositions for controlling gene expression by rna processing |
| IN2015DN01480A (da) | 2012-08-29 | 2015-07-03 | Sangamo Biosciences Inc | |
| EP3789405A1 (en) | 2012-10-12 | 2021-03-10 | The General Hospital Corporation | Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins |
| KR101706085B1 (ko) | 2012-10-23 | 2017-02-14 | 주식회사 툴젠 | 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도 |
| EP2928496B1 (en) | 2012-12-06 | 2019-10-09 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
| WO2014093479A1 (en) | 2012-12-11 | 2014-06-19 | Montana State University | Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation |
| CA2894668A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas systems and methods for altering expression of gene products in eukaryotic cells |
| ES2701749T3 (es) | 2012-12-12 | 2019-02-25 | Broad Inst Inc | Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos |
| US8993233B2 (en) | 2012-12-12 | 2015-03-31 | The Broad Institute Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| JP2016501531A (ja) | 2012-12-12 | 2016-01-21 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作および治療適用のための系、方法および組成物の送達、エンジニアリングおよび最適化 |
| CN105121648B (zh) | 2012-12-12 | 2021-05-07 | 布罗德研究所有限公司 | 用于序列操纵的系统、方法和优化的指导组合物的工程化 |
| CN105209621B (zh) | 2012-12-12 | 2021-05-25 | 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 |
| EP2784162B1 (en) | 2012-12-12 | 2015-04-08 | The Broad Institute, Inc. | Engineering of systems, methods and optimized guide compositions for sequence manipulation |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US9970024B2 (en) | 2012-12-17 | 2018-05-15 | President And Fellows Of Harvard College | RNA-guided human genome engineering |
| CA2900338A1 (en) | 2013-02-07 | 2014-08-14 | The General Hospital Corporation | Tale transcriptional activators |
| ES2901396T3 (es) | 2013-03-14 | 2022-03-22 | Caribou Biosciences Inc | Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico |
| EP2971041B1 (en) | 2013-03-15 | 2018-11-28 | 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 |
| EP3730615A3 (en) | 2013-05-15 | 2020-12-09 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| MY197877A (en) | 2013-06-04 | 2023-07-22 | Harvard College | Rna-guided transcriptional regulation |
| CA2914519A1 (en) | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| EP3011030B1 (en) | 2013-06-17 | 2023-11-08 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| EP3825406A1 (en) | 2013-06-17 | 2021-05-26 | The Broad Institute Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
| US10011850B2 (en) | 2013-06-21 | 2018-07-03 | The General Hospital Corporation | Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing |
| CN105518144A (zh) | 2013-07-09 | 2016-04-20 | 哈佛大学校长及研究员协会 | 多重rna向导的基因组工程 |
| EP3019595A4 (en) | 2013-07-09 | 2016-11-30 | THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS | |
| RU2748433C2 (ru) | 2013-07-10 | 2021-05-25 | Президент Энд Фэллоуз Оф Харвард Коллидж | ОРТОГОНАЛЬНЫЕ БЕЛКИ Cas9 ДЛЯ РНК-НАПРАВЛЯЕМОЙ РЕГУЛЯЦИИ И РЕДАКТИРОВАНИЯ ГЕНОВ |
| 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 |
| 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 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| JP6182667B2 (ja) | 2013-09-27 | 2017-08-16 | 京セラ株式会社 | エナジーセービングセルの起動のためのユーザ機器検出 |
| WO2015048690A1 (en) | 2013-09-27 | 2015-04-02 | The Regents Of The University Of California | Optimized small guide rnas and methods of use |
| US20160237455A1 (en) | 2013-09-27 | 2016-08-18 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| KR102523466B1 (ko) | 2013-11-07 | 2023-04-20 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| US20160298096A1 (en) | 2013-11-18 | 2016-10-13 | Crispr Therapeutics Ag | Crispr-cas system materials and methods |
| US9074199B1 (en) | 2013-11-19 | 2015-07-07 | President And Fellows Of Harvard College | Mutant Cas9 proteins |
| US10787684B2 (en) | 2013-11-19 | 2020-09-29 | President And Fellows Of Harvard College | Large gene excision and insertion |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| WO2015089462A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| EP3090044B1 (en) | 2013-12-26 | 2021-11-03 | The General Hospital Corporation | Multiplex guide rnas |
| WO2015134812A1 (en) | 2014-03-05 | 2015-09-11 | 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 |
| US9938521B2 (en) | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| US11339437B2 (en) * | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| WO2015188056A1 (en) | 2014-06-05 | 2015-12-10 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease design |
| JP6930834B2 (ja) | 2014-06-16 | 2021-09-01 | ザ・ジョンズ・ホプキンス・ユニバーシティー | H1プロモーターを用いるcrisprガイドrnaの発現のための組成物および方法 |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| CA2963820A1 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods 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 |
| WO2016182959A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| CN114657180A (zh) | 2015-05-16 | 2022-06-24 | 建新公司 | 深内含子突变的基因编辑 |
| CA3012607A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Crispr enzymes and systems |
| WO2017015637A1 (en) | 2015-07-22 | 2017-01-26 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
| ES2929110T3 (es) | 2015-08-25 | 2022-11-24 | Univ Duke | Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN |
| EP3344771A4 (en) | 2015-08-31 | 2019-03-20 | Agilent Technologies, Inc. | COMPOUNDS AND METHODS FOR CRISPR / CAS-BASED GENOME EDITATION THROUGH HOMOLOGOUS RECOMBINATION |
| CN110382692A (zh) | 2016-04-19 | 2019-10-25 | 博德研究所 | 新型crispr酶以及系统 |
| AU2017293773A1 (en) | 2016-07-05 | 2019-02-21 | The Johns Hopkins University | CRISPR/Cas9-based compositions and methods for treating retinal degenerations |
| CA3032822A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| WO2018126176A1 (en) | 2016-12-30 | 2018-07-05 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| EP3565907B1 (en) | 2017-01-06 | 2022-05-04 | Editas Medicine, Inc. | Methods of assessing nuclease cleavage |
-
2015
- 2015-03-10 US US14/644,181 patent/US9938521B2/en active Active
- 2015-03-10 WO PCT/US2015/019790 patent/WO2015138510A1/en not_active Ceased
- 2015-03-10 EP EP19157435.9A patent/EP3553176A1/en active Pending
- 2015-03-10 DK DK15714320.7T patent/DK3116997T3/da active
- 2015-03-10 CA CA2948728A patent/CA2948728A1/en active Pending
- 2015-03-10 EP EP15714320.7A patent/EP3116997B1/en active Active
- 2015-03-10 ES ES15714320T patent/ES2745769T3/es active Active
-
2018
- 2018-02-23 US US15/904,269 patent/US10253312B2/en active Active
-
2019
- 2019-02-19 US US16/279,941 patent/US11268086B2/en active Active
-
2022
- 2022-03-07 US US17/688,782 patent/US12234449B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| WO2015138510A1 (en) | 2015-09-17 |
| ES2745769T3 (es) | 2020-03-03 |
| US20150252358A1 (en) | 2015-09-10 |
| CA2948728A1 (en) | 2015-09-17 |
| US20230025279A1 (en) | 2023-01-26 |
| US11268086B2 (en) | 2022-03-08 |
| US20180195058A1 (en) | 2018-07-12 |
| US12234449B2 (en) | 2025-02-25 |
| US20200017849A1 (en) | 2020-01-16 |
| US10253312B2 (en) | 2019-04-09 |
| US9938521B2 (en) | 2018-04-10 |
| EP3553176A1 (en) | 2019-10-16 |
| EP3116997A1 (en) | 2017-01-18 |
| WO2015138510A8 (en) | 2016-10-06 |
| EP3116997B1 (en) | 2019-05-15 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| DK3116997T3 (da) | Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10) | |
| ZA202100808B (en) | Methods and compositions for improving plant traits | |
| IL259576A (en) | Preparations and methods for immuno-oncology | |
| IL251527A0 (en) | Preparations of adenosine deaminase-2 (ada2), their variants and methods of using them | |
| IL246951B (en) | A composition containing multiple genomes with crispr capability | |
| EP3331582A4 (en) | METHODS AND COMPOSITIONS FOR THE TREATMENT OF TRANSPLANTATION CELLS | |
| DK3102673T3 (da) | Fremgangsmåder og sammensætninger til behandling af beta-talassæmi | |
| DK3134130T3 (da) | Sammensætninger og fremgangsmåder til behandling af hæmoglobinopatier | |
| EP3337327A4 (en) | COMPOSITIONS OF NON-TOXIC PLANT AGENTS AND METHODS AND USES THEREOF | |
| DK3242957T3 (da) | Bakterieholdige sammensætninger og fremgangsmåder til anvendelse af samme til behandling og/eller forebyggelse af gastrointestinale, metaboliske og/eller andre sygdomme | |
| DK3172233T3 (da) | Anti-cd74-antistoffer, sammensætninger omfattende anti-cd74- antistoffer og fremgangsmåder til anvendelse af anti-cd74-antistoffer | |
| DK4293009T3 (da) | Fenfluraminsammensætninger og fremgangsmåder til fremstilling af samme | |
| DK3371313T3 (da) | Fremgangsmåder og sammensætninger til forbedret plantetransformation | |
| 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 | |
| IL249640A0 (en) | Preparations for the treatment of nosebleeds | |
| EP3204426A4 (en) | Compositions and methods for treating viral hemorrhagic fever | |
| 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 |