WO2024006770A1 - Compositions et procédés pour le traitement de dystrophies myotoniques - Google Patents
Compositions et procédés pour le traitement de dystrophies myotoniques Download PDFInfo
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- 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
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- 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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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
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- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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Abstract
La présente invention concerne l'administration d'un virus adéno-associé recombinant (rAAV) de polynucléotides pour le traitement de l'épissage anormal et de troubles de la dominance de l'ARN, y compris des troubles caractérisés par la rétention nucléaire de transcrits d'ARN contenant des régions de répétition étendues de manière aberrante qui se lient et des protéines de facteur d'épissage séquentielles. L'invention concerne des produits rAAV et des procédés d'utilisation du rAAV dans le traitement de la dystrophie myotonique.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263355891P | 2022-06-27 | 2022-06-27 | |
| US63/355,891 | 2022-06-27 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| WO2024006770A1 true WO2024006770A1 (fr) | 2024-01-04 |
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ID=89381657
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PCT/US2023/069184 WO2024006770A1 (fr) | 2022-06-27 | 2023-06-27 | Compositions et procédés pour le traitement de dystrophies myotoniques |
Country Status (1)
| Country | Link |
|---|---|
| WO (1) | WO2024006770A1 (fr) |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019222354A1 (fr) * | 2018-05-15 | 2019-11-21 | University Of Washington | Compositions et procédés pour réduire les anomalies d'épissage et traiter des troubles de dominance arn |
| US10526617B2 (en) * | 2001-11-13 | 2020-01-07 | The Trustees Of The University Of Pennsylvania | Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby |
| US20220033823A1 (en) * | 2018-08-22 | 2022-02-03 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene |
-
2023
- 2023-06-27 WO PCT/US2023/069184 patent/WO2024006770A1/fr unknown
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10526617B2 (en) * | 2001-11-13 | 2020-01-07 | The Trustees Of The University Of Pennsylvania | Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby |
| WO2019222354A1 (fr) * | 2018-05-15 | 2019-11-21 | University Of Washington | Compositions et procédés pour réduire les anomalies d'épissage et traiter des troubles de dominance arn |
| US20220033823A1 (en) * | 2018-08-22 | 2022-02-03 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene |
Non-Patent Citations (1)
| Title |
|---|
| IZZO MARIAPAOLA, BATTISTINI JONATHAN, PROVENZANO CLAUDIA, MARTELLI FABIO, CARDINALI BEATRICE, FALCONE GERMANA: "Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing", INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, MOLECULAR DIVERSITY PRESERVATION INTERNATIONAL (MDPI), BASEL, CH, vol. 23, no. 9, 21 April 2022 (2022-04-21), Basel, CH , pages 4622, XP093127491, ISSN: 1422-0067, DOI: 10.3390/ijms23094622 * |
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