CA3048635A1 - Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences - Google Patents
Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences Download PDFInfo
- Publication number
- CA3048635A1 CA3048635A1 CA3048635A CA3048635A CA3048635A1 CA 3048635 A1 CA3048635 A1 CA 3048635A1 CA 3048635 A CA3048635 A CA 3048635A CA 3048635 A CA3048635 A CA 3048635A CA 3048635 A1 CA3048635 A1 CA 3048635A1
- Authority
- CA
- Canada
- Prior art keywords
- sequence encoding
- promoter
- sequence
- nucleic acid
- vector
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
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
- C12N15/63—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- 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
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/0657—Cardiomyocytes; Heart cells
-
- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/0658—Skeletal muscle cells, e.g. myocytes, myotubes, myoblasts
- C12N5/0659—Satellite cells
-
- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/066—Tenocytes; Tendons, Ligaments
-
- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/0661—Smooth muscle cells
-
- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
-
- 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
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- C12N2510/00—Genetically modified cells
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/90—Vectors containing a transposable element
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Cell Biology (AREA)
- Plant Pathology (AREA)
- Rheumatology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Transplantation (AREA)
- Developmental Biology & Embryology (AREA)
- Virology (AREA)
- Mycology (AREA)
- Crystallography & Structural Chemistry (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762442606P | 2017-01-05 | 2017-01-05 | |
| US62/442,606 | 2017-01-05 | ||
| US201762544449P | 2017-08-11 | 2017-08-11 | |
| US62/544,449 | 2017-08-11 | ||
| US201762596298P | 2017-12-08 | 2017-12-08 | |
| US62/596,298 | 2017-12-08 | ||
| PCT/US2018/012558 WO2018129296A1 (en) | 2017-01-05 | 2018-01-05 | Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3048635A1 true CA3048635A1 (en) | 2018-07-12 |
Family
ID=61193018
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3048635A Pending CA3048635A1 (en) | 2017-01-05 | 2018-01-05 | Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US20190338311A1 (https=) |
| EP (1) | EP3565897B1 (https=) |
| JP (1) | JP2020503869A (https=) |
| KR (1) | KR102606174B1 (https=) |
| CN (1) | CN110506115A (https=) |
| AU (1) | AU2018205521A1 (https=) |
| BR (1) | BR112019013962A2 (https=) |
| CA (1) | CA3048635A1 (https=) |
| CL (1) | CL2019001882A1 (https=) |
| CO (1) | CO2019008181A2 (https=) |
| CR (1) | CR20190326A (https=) |
| DK (1) | DK3565897T3 (https=) |
| DO (1) | DOP2019000179A (https=) |
| EC (1) | ECSP19056408A (https=) |
| IL (1) | IL267786A (https=) |
| JO (1) | JOP20190166A1 (https=) |
| MA (1) | MA47239A (https=) |
| MX (1) | MX2019008064A (https=) |
| PE (1) | PE20191357A1 (https=) |
| PH (1) | PH12019501561A1 (https=) |
| SG (1) | SG11201906147VA (https=) |
| WO (1) | WO2018129296A1 (https=) |
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| 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 |
| EP3617311B1 (en) * | 2017-03-30 | 2024-11-20 | Kyoto University | Method for inducing exon skipping by genome editing |
| EP3735462A1 (en) * | 2018-01-05 | 2020-11-11 | The Board of Regents of The University of Texas System | Therapeutic crispr/cas9 compositions and methods of use |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| EP3810775A1 (en) * | 2018-06-21 | 2021-04-28 | The Board Of Regents Of The University Of Texas System | Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020122104A1 (ja) | 2018-12-11 | 2020-06-18 | 国立大学法人京都大学 | ゲノムdnaに欠失を誘導する方法 |
| WO2020142714A1 (en) * | 2019-01-04 | 2020-07-09 | Exonics Therapeutics, Inc. | Aav expression cassette and aav vectors comprising the same |
| BR112021017655A2 (pt) * | 2019-03-07 | 2021-11-16 | Univ Columbia | Método e sistema de integração de dna guiada por rna usando transposons tipo tn7 |
| US20220177876A1 (en) * | 2019-04-12 | 2022-06-09 | The Regents Of The University Of California | Compositions and Methods for Modifying Dystrophin Genes |
| JP2022526669A (ja) * | 2019-04-12 | 2022-05-25 | デューク ユニバーシティ | ジストロフィン機能を修復するためのCRISPR/Casをベースにした塩基編集組成物 |
| US20210047649A1 (en) | 2019-05-08 | 2021-02-18 | Vertex Pharmaceuticals Incorporated | Crispr/cas all-in-two vector systems for treatment of dmd |
| KR102264115B1 (ko) * | 2019-05-10 | 2021-06-14 | 한국과학기술연구원 | 무-운반체 다중 CRISPR/Cas 9 유전자 편집 복합체 및 그의 용도 |
| CN110499333A (zh) * | 2019-08-01 | 2019-11-26 | 广州德赫生物科技有限公司 | 用于修复dmd基因突变的核酸序列及系统 |
| WO2021072276A1 (en) * | 2019-10-11 | 2021-04-15 | Yale University | Compositions and methods for upregulating isoforms of dystrophin as therapy for duchenne muscular dystrophy (dmd) |
| CN114846146B (zh) * | 2019-10-29 | 2024-04-12 | 基恩科雷有限责任公司 | 用于增加CRISPR/Cas12f1系统的效率的工程化引导RNA及其用途 |
| EP4103214B1 (en) * | 2020-02-13 | 2026-04-29 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| CN111172191B (zh) * | 2020-02-21 | 2020-12-22 | 浙江大学 | 一种高效基因敲除载体及其应用 |
| EP4125349A4 (en) * | 2020-04-27 | 2024-07-10 | Duke University | GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS |
| CN115011598A (zh) * | 2020-09-02 | 2022-09-06 | 西湖大学 | 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用 |
| AU2021342057A1 (en) * | 2020-09-09 | 2023-04-06 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of duchenne muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| JP2024525608A (ja) | 2021-07-09 | 2024-07-12 | ダイン セラピューティクス,インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体および製剤 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2023159103A1 (en) * | 2022-02-17 | 2023-08-24 | The Board Of Regents Of The University Of Texas System | CRISPR/SpCas9 VARIANT AND METHODS FOR ENHANCED CORRECTON OF DUCHENNE MUSCULAR DYSTROPHY MUTATIONS |
| KR20230134098A (ko) * | 2022-03-10 | 2023-09-20 | 주식회사 진코어 | 듀센 근이영양증 치료를 위한 유전자 편집 시스템 및 이를 이용한 질병 치료 방법 |
| CN115820642B (zh) * | 2022-11-11 | 2023-10-10 | 昆明理工大学 | 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK1737966T3 (da) * | 2004-04-02 | 2012-07-09 | Univ Texas | Cancerspecifik promotorer |
| 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 |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| EP4659765A3 (en) * | 2014-03-12 | 2026-02-18 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| CN106714845A (zh) * | 2014-08-11 | 2017-05-24 | 得克萨斯州大学系统董事会 | 通过crispr/cas9介导的基因编辑预防肌营养不良 |
| US20170266320A1 (en) * | 2014-12-01 | 2017-09-21 | President And Fellows Of Harvard College | RNA-Guided Systems for In Vivo Gene Editing |
| JP6832280B2 (ja) * | 2015-01-16 | 2021-02-24 | ユニバーシティ オブ ワシントンUniversity of Washington | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| 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 |
| WO2016174056A1 (en) * | 2015-04-27 | 2016-11-03 | Genethon | Compositions and methods for the treatment of nucleotide repeat expansion disorders |
| AU2016344609B2 (en) * | 2015-10-28 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of duchenne muscular dystrophy |
| KR102787119B1 (ko) * | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| US20170362635A1 (en) * | 2016-06-20 | 2017-12-21 | University Of Washington | Muscle-specific crispr/cas9 editing of genes |
-
2017
- 2017-06-16 JO JOP/2019/0166A patent/JOP20190166A1/ar unknown
-
2018
- 2018-01-05 WO PCT/US2018/012558 patent/WO2018129296A1/en not_active Ceased
- 2018-01-05 SG SG11201906147VA patent/SG11201906147VA/en unknown
- 2018-01-05 MX MX2019008064A patent/MX2019008064A/es unknown
- 2018-01-05 MA MA047239A patent/MA47239A/fr unknown
- 2018-01-05 EP EP18704637.0A patent/EP3565897B1/en active Active
- 2018-01-05 CN CN201880010288.7A patent/CN110506115A/zh active Pending
- 2018-01-05 CR CR20190326A patent/CR20190326A/es unknown
- 2018-01-05 BR BR112019013962-1A patent/BR112019013962A2/pt not_active IP Right Cessation
- 2018-01-05 PE PE2019001384A patent/PE20191357A1/es unknown
- 2018-01-05 CA CA3048635A patent/CA3048635A1/en active Pending
- 2018-01-05 KR KR1020197022753A patent/KR102606174B1/ko active Active
- 2018-01-05 DK DK18704637.0T patent/DK3565897T3/da active
- 2018-01-05 JP JP2019536508A patent/JP2020503869A/ja active Pending
- 2018-01-05 US US16/476,137 patent/US20190338311A1/en not_active Abandoned
- 2018-01-05 AU AU2018205521A patent/AU2018205521A1/en not_active Abandoned
-
2019
- 2019-07-02 IL IL267786A patent/IL267786A/en unknown
- 2019-07-02 PH PH12019501561A patent/PH12019501561A1/en unknown
- 2019-07-05 CL CL2019001882A patent/CL2019001882A1/es unknown
- 2019-07-05 DO DO2019000179A patent/DOP2019000179A/es unknown
- 2019-07-29 CO CO2019008181A patent/CO2019008181A2/es unknown
- 2019-08-06 EC ECSENADI201956408A patent/ECSP19056408A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| DK3565897T3 (da) | 2025-02-10 |
| IL267786A (en) | 2019-09-26 |
| CN110506115A (zh) | 2019-11-26 |
| EP3565897B1 (en) | 2024-12-11 |
| CO2019008181A2 (es) | 2019-10-31 |
| KR102606174B1 (ko) | 2023-11-27 |
| MX2019008064A (es) | 2020-07-20 |
| JP2020503869A (ja) | 2020-02-06 |
| EP3565897A1 (en) | 2019-11-13 |
| ECSP19056408A (es) | 2019-09-30 |
| KR20190100967A (ko) | 2019-08-29 |
| PE20191357A1 (es) | 2019-10-01 |
| SG11201906147VA (en) | 2019-08-27 |
| BR112019013962A2 (pt) | 2020-02-11 |
| CR20190326A (es) | 2019-10-02 |
| JOP20190166A1 (ar) | 2019-07-02 |
| CL2019001882A1 (es) | 2019-10-04 |
| PH12019501561A1 (en) | 2019-09-09 |
| US20190338311A1 (en) | 2019-11-07 |
| AU2018205521A1 (en) | 2019-07-18 |
| WO2018129296A1 (en) | 2018-07-12 |
| DOP2019000179A (es) | 2019-11-15 |
| MA47239A (fr) | 2019-11-13 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| EP3565897B1 (en) | Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences | |
| JP7197617B2 (ja) | Crispr/cas9媒介遺伝子編集による筋ジストロフィーの予防 | |
| US20200046854A1 (en) | Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing | |
| US20200370042A1 (en) | Compositions and methods for correcting dystrophin mutations in human cardiomyocytes | |
| US20190364862A1 (en) | Dmd reporter models containing humanized duchenne muscular dystrophy mutations | |
| US20200260698A1 (en) | Exon deletion correction of duchenne muscular dystrophy mutations in the dystrophin actin binding domain 1 using crispr genome editing | |
| US20210261962A1 (en) | Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy | |
| EP3735462A1 (en) | Therapeutic crispr/cas9 compositions and methods of use | |
| OA20296A (en) | Optimized strategy for exon skipping modifications using CRISPR/CAS9 with triple guide sequences. | |
| Class et al. | Patent application title: PREVENTION OF MUSCULAR DYSTROPHY BY CRISPR/CAS9-MEDIATED GENE EDITING Inventors: Eric N. Olson (Dallas, TX, US) Eric N. Olson (Dallas, TX, US) Chengzu Long (Dallas, TX, US) John R. Mcanally (Dallas, TX, US) John M. Shelton (Dallas, TX, US) Rhonda Bassel-Duby (Dallas, TX, US) |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20230104 |
|
| U13 | Renewal or maintenance fee not paid |
Free format text: ST27 STATUS EVENT CODE: N-2-6-U10-U13-U300 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO MAINTENANCE FEE NOTICE Effective date: 20240705 |
|
| N11 | Application terminated |
Free format text: ST27 STATUS EVENT CODE: T-6-6-N10-N11-N106 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION NOT REINSTATED BY DEADLINE Effective date: 20250707 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: N-6-6-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20251229 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: N-6-6-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20260216 |