CL2021002680A1 - Deleción mediada por vector aav de punto activo mutacional grande para el tratamiento de distrofia muscular de duchenne - Google Patents
Deleción mediada por vector aav de punto activo mutacional grande para el tratamiento de distrofia muscular de duchenneInfo
- Publication number
- CL2021002680A1 CL2021002680A1 CL2021002680A CL2021002680A CL2021002680A1 CL 2021002680 A1 CL2021002680 A1 CL 2021002680A1 CL 2021002680 A CL2021002680 A CL 2021002680A CL 2021002680 A CL2021002680 A CL 2021002680A CL 2021002680 A1 CL2021002680 A1 CL 2021002680A1
- Authority
- CL
- Chile
- Prior art keywords
- muscular dystrophy
- aav vector
- duchenne muscular
- active point
- mediated deletion
- Prior art date
Links
Classifications
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- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- 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
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- 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
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding 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
- C12N15/63—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
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- 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
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- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2800/40—Systems of functionally co-operating vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Crystallography & Structural Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962833760P | 2019-04-14 | 2019-04-14 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2021002680A1 true CL2021002680A1 (es) | 2022-07-15 |
Family
ID=72837591
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2021002680A CL2021002680A1 (es) | 2019-04-14 | 2021-10-13 | Deleción mediada por vector aav de punto activo mutacional grande para el tratamiento de distrofia muscular de duchenne |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US20220184229A1 (https=) |
| EP (1) | EP3931331A4 (https=) |
| JP (2) | JP2022529631A (https=) |
| KR (1) | KR20210151916A (https=) |
| CN (1) | CN113785063A (https=) |
| AR (1) | AR118670A1 (https=) |
| AU (1) | AU2020257898B2 (https=) |
| BR (1) | BR112021020515A2 (https=) |
| CA (1) | CA3137248A1 (https=) |
| CL (1) | CL2021002680A1 (https=) |
| CO (1) | CO2021013692A2 (https=) |
| EA (1) | EA202192815A1 (https=) |
| IL (1) | IL287163A (https=) |
| MX (1) | MX2021012511A (https=) |
| SG (1) | SG11202111172QA (https=) |
| TW (2) | TWI887234B (https=) |
| WO (1) | WO2020214609A1 (https=) |
Families Citing this family (16)
| 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 |
| WO2017035416A2 (en) | 2015-08-25 | 2017-03-02 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
| 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 |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| US20230272428A1 (en) * | 2019-12-16 | 2023-08-31 | President And Fellows Of Harvard College | Methods and compositions for correction of dmd mutations |
| US20230346978A1 (en) * | 2020-04-14 | 2023-11-02 | University Of Massachusetts | Dcas13-mediated therapeutic rna base editing for in vivo gene therapy |
| WO2022087321A1 (en) * | 2020-10-21 | 2022-04-28 | Duke University | Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy |
| MX2024002988A (es) * | 2021-09-08 | 2024-06-11 | Flagship Pioneering Innovations Vi Llc | Composiciones y metodos moduladores de la serpina. |
| IL317874A (en) | 2022-06-24 | 2025-02-01 | Tune Therapeutics Inc | Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression |
| WO2024193704A1 (en) * | 2023-03-22 | 2024-09-26 | Huidagene Therapeutics Co., Ltd. | Guide nucleic acids targeting dmd and uses thereof |
| WO2025221969A2 (en) | 2024-04-18 | 2025-10-23 | Sarepta Therapeutics, Inc. | Genetic constructs for gene editing |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2005035718A2 (en) * | 2003-10-03 | 2005-04-21 | Welgen, Inc. | Biderectional promoters for small rna expression |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| 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 |
| 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 |
| EP3199632A1 (en) * | 2016-01-26 | 2017-08-02 | ACIB GmbH | Temperature-inducible crispr/cas system |
| WO2017139505A2 (en) * | 2016-02-11 | 2017-08-17 | The Regents Of The University Of California | Methods and compositions for modifying a mutant dystrophin gene in a cell's genome |
| US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
| US10687520B2 (en) * | 2017-03-07 | 2020-06-23 | The Board Of Regents Of The University Of Texas System | Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44 |
| US11779653B2 (en) * | 2017-09-29 | 2023-10-10 | The Regents Of The University Of California | Multi-armed polyrotaxane platform for protected nucleic acid delivery |
-
2020
- 2020-04-14 WO PCT/US2020/028148 patent/WO2020214609A1/en not_active Ceased
- 2020-04-14 MX MX2021012511A patent/MX2021012511A/es unknown
- 2020-04-14 CA CA3137248A patent/CA3137248A1/en active Pending
- 2020-04-14 US US17/603,330 patent/US20220184229A1/en active Pending
- 2020-04-14 AR ARP200101048A patent/AR118670A1/es unknown
- 2020-04-14 EP EP20790851.8A patent/EP3931331A4/en active Pending
- 2020-04-14 AU AU2020257898A patent/AU2020257898B2/en active Active
- 2020-04-14 SG SG11202111172QA patent/SG11202111172QA/en unknown
- 2020-04-14 CN CN202080028248.2A patent/CN113785063A/zh active Pending
- 2020-04-14 TW TW109112555A patent/TWI887234B/zh active
- 2020-04-14 TW TW114119381A patent/TW202536186A/zh unknown
- 2020-04-14 BR BR112021020515A patent/BR112021020515A2/pt unknown
- 2020-04-14 JP JP2021560849A patent/JP2022529631A/ja active Pending
- 2020-04-14 KR KR1020217036783A patent/KR20210151916A/ko active Pending
- 2020-04-14 EA EA202192815A patent/EA202192815A1/ru unknown
-
2021
- 2021-10-11 IL IL287163A patent/IL287163A/en unknown
- 2021-10-13 CL CL2021002680A patent/CL2021002680A1/es unknown
- 2021-10-14 CO CONC2021/0013692A patent/CO2021013692A2/es unknown
-
2025
- 2025-01-31 JP JP2025014814A patent/JP2025087680A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| TW202104594A (zh) | 2021-02-01 |
| IL287163A (en) | 2021-12-01 |
| AR118670A1 (es) | 2021-10-20 |
| EP3931331A1 (en) | 2022-01-05 |
| EA202192815A1 (ru) | 2022-01-24 |
| CN113785063A (zh) | 2021-12-10 |
| US20220184229A1 (en) | 2022-06-16 |
| AU2020257898B2 (en) | 2026-03-05 |
| WO2020214609A1 (en) | 2020-10-22 |
| JP2022529631A (ja) | 2022-06-23 |
| AU2020257898A1 (en) | 2021-11-04 |
| BR112021020515A2 (pt) | 2022-01-04 |
| SG11202111172QA (en) | 2021-11-29 |
| KR20210151916A (ko) | 2021-12-14 |
| CA3137248A1 (en) | 2020-10-22 |
| CO2021013692A2 (es) | 2022-01-17 |
| TW202536186A (zh) | 2025-09-16 |
| JP2025087680A (ja) | 2025-06-10 |
| TWI887234B (zh) | 2025-06-21 |
| MX2021012511A (es) | 2022-01-06 |
| EP3931331A4 (en) | 2022-11-30 |
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