IL307305A - Preparations and methods for the treatment of TDP-43 proteinopathy - Google Patents
Preparations and methods for the treatment of TDP-43 proteinopathyInfo
- Publication number
- IL307305A IL307305A IL307305A IL30730523A IL307305A IL 307305 A IL307305 A IL 307305A IL 307305 A IL307305 A IL 307305A IL 30730523 A IL30730523 A IL 30730523A IL 307305 A IL307305 A IL 307305A
- Authority
- IL
- Israel
- Prior art keywords
- seq
- exon
- cryptic
- unc13a
- antisense oligonucleotide
- Prior art date
Links
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- 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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- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
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- C12N2320/33—Alteration of splicing
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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| US202163171522P | 2021-04-06 | 2021-04-06 | |
| US202263312808P | 2022-02-22 | 2022-02-22 | |
| PCT/US2022/023559 WO2022216759A1 (en) | 2021-04-06 | 2022-04-05 | Compositions and methods for treating tdp-43 proteinopathy |
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| CA3227115A1 (en) * | 2021-07-21 | 2023-01-26 | Wen-Hsuan Chang | Unc13a antisense oligonucleotides |
| CN119790152A (zh) * | 2021-12-03 | 2025-04-08 | 奎里斯公司 | 使用unc13a基因转录本的调节剂治疗神经系统疾病 |
| GB202117758D0 (en) * | 2021-12-09 | 2022-01-26 | Ucl Business Ltd | Therapeutics for the treatment of neurodegenerative disorders |
| CN118974254A (zh) * | 2021-12-21 | 2024-11-15 | 豪夫迈·罗氏有限公司 | 靶向unc13a的反义寡核苷酸 |
| AU2023356317A1 (en) * | 2022-10-05 | 2025-05-15 | Trace Neuroscience, Inc. | Unc13a antisense oligonucleotides and uses thereof |
| WO2024155986A2 (en) * | 2023-01-20 | 2024-07-25 | AcuraStem Incorporated | Unc13a antisense oligonucleotides |
| WO2024178223A1 (en) * | 2023-02-24 | 2024-08-29 | Northwestern University | Antisense oligonucleotides for preventing unc13a misplicing |
| AU2024283557A1 (en) * | 2023-06-02 | 2026-01-08 | Quralis Corporation | Modified unc13a oligonucleotides |
| GB202309922D0 (en) * | 2023-06-29 | 2023-08-16 | Univ Edinburgh | Antisense treatment |
| WO2025007194A1 (en) * | 2023-07-05 | 2025-01-09 | Macquarie University | Modulation of gene expression |
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| EP4410805A3 (en) | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| BR112018012894A2 (en) | 2015-12-23 | 2018-12-04 | Crispr Therapeutics Ag | Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration |
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| CA3079727A1 (en) | 2017-10-24 | 2019-05-02 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of rare diseases |
| JP7795914B2 (ja) * | 2019-06-03 | 2026-01-08 | クラリス コーポレーション | 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法 |
| WO2022018187A1 (en) * | 2020-07-23 | 2022-01-27 | F. Hoffmann-La Roche Ag | Oligonucleotides targeting rna binding protein sites |
| GB2603454A (en) * | 2020-12-09 | 2022-08-10 | Ucl Business Ltd | Novel therapeutics for the treatment of neurodegenerative disorders |
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| US20250011773A1 (en) | 2025-01-09 |
| CA3213590A1 (en) | 2022-10-13 |
| MX2023011794A (es) | 2024-01-08 |
| EP4320236A1 (en) | 2024-02-14 |
| JP2024513237A (ja) | 2024-03-22 |
| KR20240004467A (ko) | 2024-01-11 |
| WO2022216759A1 (en) | 2022-10-13 |
| AU2022255175A1 (en) | 2023-11-23 |
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