BR112022020945A2 - Inibição por crispr para distrofia muscular fácioescápulo- umeral - Google Patents
Inibição por crispr para distrofia muscular fácioescápulo- umeralInfo
- Publication number
- BR112022020945A2 BR112022020945A2 BR112022020945A BR112022020945A BR112022020945A2 BR 112022020945 A2 BR112022020945 A2 BR 112022020945A2 BR 112022020945 A BR112022020945 A BR 112022020945A BR 112022020945 A BR112022020945 A BR 112022020945A BR 112022020945 A2 BR112022020945 A2 BR 112022020945A2
- Authority
- BR
- Brazil
- Prior art keywords
- muscular dystrophy
- facioscapulohumeral muscular
- crispr
- dux4
- inhibition
- Prior art date
Links
Classifications
-
- 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
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
-
- 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
- C12N2750/14141—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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Bakery Products And Manufacturing Methods Therefor (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063011476P | 2020-04-17 | 2020-04-17 | |
| PCT/US2021/025940 WO2021211325A1 (en) | 2020-04-17 | 2021-04-06 | Crispr-inhibition for facioscapulohumeral muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022020945A2 true BR112022020945A2 (pt) | 2022-12-27 |
Family
ID=78084611
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022020945A BR112022020945A2 (pt) | 2020-04-17 | 2021-04-06 | Inibição por crispr para distrofia muscular fácioescápulo- umeral |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230174958A1 (https=) |
| EP (1) | EP4135778A4 (https=) |
| JP (1) | JP2023522020A (https=) |
| KR (1) | KR20230003511A (https=) |
| CN (1) | CN115768487A (https=) |
| AU (1) | AU2021257213A1 (https=) |
| BR (1) | BR112022020945A2 (https=) |
| CA (1) | CA3175625A1 (https=) |
| IL (1) | IL297113A (https=) |
| MX (1) | MX2022012965A (https=) |
| WO (1) | WO2021211325A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20230023612A (ko) | 2020-04-02 | 2023-02-17 | 마이레큘, 인크. | 조작된 올리고뉴클레오티드를 사용한 표적화된 억제 |
| WO2022045366A1 (en) * | 2020-08-31 | 2022-03-03 | Modalis Therapeutics Corporation | Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene |
| WO2024020444A2 (en) * | 2022-07-20 | 2024-01-25 | Nevada Research & Innovation Corporation | Muscle-specific regulatory cassettes |
| WO2025019820A1 (en) * | 2023-07-19 | 2025-01-23 | Nevada Research & Innovation Corporation | All in one vectors for the treatment of facioscapulohumeral muscular dystrophy |
| CN117448380A (zh) * | 2023-12-22 | 2024-01-26 | 上海元戊医学技术有限公司 | iPSC来源的COL10A1蛋白低表达MSC细胞株的构建方法及其应用 |
| WO2026039783A1 (en) * | 2024-08-16 | 2026-02-19 | Sarepta Therapeutics, Inc. | Deficient cas-transcriptional repressor fusions for regulation of gene expression |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3192880B1 (en) * | 2010-08-18 | 2019-10-09 | Fred Hutchinson Cancer Research Center | Nucleic acid agents for use in treating facioscapulohumeral dystrophy (fshd) |
| JP7103750B2 (ja) * | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用 |
| PL3097197T3 (pl) * | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
| EP3515506B1 (en) * | 2016-09-23 | 2023-09-13 | University of Massachusetts | Silencing of dux4 by recombinant gene editing complexes |
| US11160823B2 (en) * | 2016-11-07 | 2021-11-02 | University Of Massachusetts | Therapeutic targets for facioscapulohumeral muscular dystrophy |
| WO2019051290A1 (en) * | 2017-09-07 | 2019-03-14 | The Children's Medical Center Corporation | COMPOSITIONS AND METHODS FOR THE TREATMENT OF FACIO-SCAPULO-HUMERAL DYSTROPHY |
| BR112020006677A2 (pt) * | 2017-10-05 | 2020-10-06 | Fulcrum Therapeutics, Inc. | uso de inibidores p38 para reduzir a expressão de dux4 |
-
2021
- 2021-04-06 AU AU2021257213A patent/AU2021257213A1/en active Pending
- 2021-04-06 CN CN202180041592.XA patent/CN115768487A/zh active Pending
- 2021-04-06 JP JP2022562629A patent/JP2023522020A/ja active Pending
- 2021-04-06 CA CA3175625A patent/CA3175625A1/en active Pending
- 2021-04-06 KR KR1020227039447A patent/KR20230003511A/ko active Pending
- 2021-04-06 WO PCT/US2021/025940 patent/WO2021211325A1/en not_active Ceased
- 2021-04-06 BR BR112022020945A patent/BR112022020945A2/pt unknown
- 2021-04-06 IL IL297113A patent/IL297113A/en unknown
- 2021-04-06 US US17/919,198 patent/US20230174958A1/en active Pending
- 2021-04-06 MX MX2022012965A patent/MX2022012965A/es unknown
- 2021-04-06 EP EP21787712.5A patent/EP4135778A4/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023522020A (ja) | 2023-05-26 |
| EP4135778A4 (en) | 2024-05-29 |
| IL297113A (en) | 2022-12-01 |
| US20230174958A1 (en) | 2023-06-08 |
| CN115768487A (zh) | 2023-03-07 |
| AU2021257213A1 (en) | 2022-11-03 |
| MX2022012965A (es) | 2023-01-18 |
| CA3175625A1 (en) | 2021-10-21 |
| EP4135778A1 (en) | 2023-02-22 |
| KR20230003511A (ko) | 2023-01-06 |
| WO2021211325A1 (en) | 2021-10-21 |
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