RU2749971C2 - Средство лечения болезни гентингтона на основе aav - Google Patents
Средство лечения болезни гентингтона на основе aav Download PDFInfo
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- RU2749971C2 RU2749971C2 RU2019111648A RU2019111648A RU2749971C2 RU 2749971 C2 RU2749971 C2 RU 2749971C2 RU 2019111648 A RU2019111648 A RU 2019111648A RU 2019111648 A RU2019111648 A RU 2019111648A RU 2749971 C2 RU2749971 C2 RU 2749971C2
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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
- 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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- 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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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
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- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
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- C12N2750/14011—Parvoviridae
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- C12N2750/14011—Parvoviridae
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- 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
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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- Health & Medical Sciences (AREA)
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- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Psychology (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662398487P | 2016-09-22 | 2016-09-22 | |
| US62/398,487 | 2016-09-22 | ||
| PCT/US2017/052902 WO2018057855A1 (en) | 2016-09-22 | 2017-09-22 | Aav treatment of huntington's disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2019111648A RU2019111648A (ru) | 2020-10-22 |
| RU2019111648A3 RU2019111648A3 (enExample) | 2020-11-23 |
| RU2749971C2 true RU2749971C2 (ru) | 2021-06-21 |
Family
ID=61691085
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2019111648A RU2749971C2 (ru) | 2016-09-22 | 2017-09-22 | Средство лечения болезни гентингтона на основе aav |
Country Status (19)
| Country | Link |
|---|---|
| US (5) | US10457940B2 (enExample) |
| EP (1) | EP3515505A4 (enExample) |
| JP (3) | JP6884430B2 (enExample) |
| KR (1) | KR102271675B1 (enExample) |
| CN (2) | CN117025677A (enExample) |
| AU (2) | AU2017330411B2 (enExample) |
| BR (2) | BR112019005548B1 (enExample) |
| CA (1) | CA3037929A1 (enExample) |
| CL (1) | CL2019000732A1 (enExample) |
| CO (1) | CO2019003846A2 (enExample) |
| IL (3) | IL308514B2 (enExample) |
| MX (2) | MX2019003376A (enExample) |
| MY (1) | MY195386A (enExample) |
| PE (2) | PE20241233A1 (enExample) |
| PH (1) | PH12019500626A1 (enExample) |
| RU (1) | RU2749971C2 (enExample) |
| SA (1) | SA519401379B1 (enExample) |
| WO (1) | WO2018057855A1 (enExample) |
| ZA (1) | ZA201902226B (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| MX2019013172A (es) * | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| WO2019159884A1 (ja) * | 2018-02-13 | 2019-08-22 | 東レ株式会社 | 認知症の検出のためのキット又はデバイス及び方法 |
| US11649455B2 (en) | 2018-03-30 | 2023-05-16 | University Of Geneva | Micro RNA expression constructs and uses thereof |
| WO2019213128A1 (en) | 2018-04-30 | 2019-11-07 | The Brigham And Women's Hospital, Inc. | Compositions and therapeutics methods of microrna gene delivery |
| WO2019222413A1 (en) | 2018-05-15 | 2019-11-21 | University Of Massachusetts | Modified aav constructs and uses thereof |
| US12391947B2 (en) | 2018-11-29 | 2025-08-19 | University Of Massachusetts | Modulation of SPTLC1 via recombinant adeno-associated vectors |
| EP3908658A1 (en) * | 2019-01-09 | 2021-11-17 | Universidade de Coimbra | Double stranded rna and uses thereof |
| WO2021087036A1 (en) * | 2019-11-01 | 2021-05-06 | Alnylam Pharmaceuticals, Inc. | HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
| JP7730327B2 (ja) * | 2019-12-20 | 2025-08-27 | アスクバイオ インコーポレイテッド | ハンチントン病を処置するための方法 |
| US20230365963A1 (en) * | 2020-09-21 | 2023-11-16 | Asklepios Biopharmaceutical, Inc. | Methods for treating neurological disease |
| CN117320741A (zh) * | 2020-12-01 | 2023-12-29 | 洛卡纳生物股份有限公司 | 用于治疗cag重复疾病的靶向rna的组合物和方法 |
| WO2022155364A1 (en) * | 2021-01-13 | 2022-07-21 | Dignity Health | Modulation of chitinase protein expression |
| EP4317434A4 (en) * | 2021-03-30 | 2025-04-16 | Nanjing University | RNA DELIVERY SYSTEM FOR THE TREATMENT OF HUNTINGTON'S DISEASE |
| AU2023211606A1 (en) * | 2022-01-27 | 2024-07-11 | AskBio Inc. | Compositions for treating neurological disease |
| CN115487315B (zh) * | 2022-04-20 | 2023-08-04 | 暨南大学 | 治疗亨廷顿病的药物 |
| US20240360443A1 (en) | 2023-04-09 | 2024-10-31 | Dystrogen Gene Therapies Inc. | Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats |
| WO2025027116A1 (en) | 2023-08-01 | 2025-02-06 | Institut Curie | Nanoparticles comprising nucleic acid sequences encoding cyclic gmp-amp synthase |
| CN122319242A (zh) * | 2023-10-11 | 2026-06-30 | 优尼科生物制药有限公司 | 亨廷顿蛋白基因的新型核酸调节 |
| WO2025160434A1 (en) * | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting huntington's disease |
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| EA023938B1 (ru) * | 2009-04-30 | 2016-07-29 | Оспедале Сан Рафаэлле С.Р.Л. | Вектор гена |
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