JP7327803B2 - 筋萎縮性側索硬化症(als)を処置する方法 - Google Patents
筋萎縮性側索硬化症(als)を処置する方法 Download PDFInfo
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- JP7327803B2 JP7327803B2 JP2019562332A JP2019562332A JP7327803B2 JP 7327803 B2 JP7327803 B2 JP 7327803B2 JP 2019562332 A JP2019562332 A JP 2019562332A JP 2019562332 A JP2019562332 A JP 2019562332A JP 7327803 B2 JP7327803 B2 JP 7327803B2
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- 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
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- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- A01K2217/00—Genetically modified animals
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- A01K2267/03—Animal model, e.g. for test or diseases
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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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
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Biodiversity & Conservation Biology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762503909P | 2017-05-09 | 2017-05-09 | |
| US62/503,909 | 2017-05-09 | ||
| PCT/US2018/031880 WO2018208972A1 (en) | 2017-05-09 | 2018-05-09 | Methods of treating amyotrophic lateral sclerosis (als) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020519284A JP2020519284A (ja) | 2020-07-02 |
| JP2020519284A5 JP2020519284A5 (https=) | 2021-07-26 |
| JP7327803B2 true JP7327803B2 (ja) | 2023-08-16 |
Family
ID=64104908
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019562332A Active JP7327803B2 (ja) | 2017-05-09 | 2018-05-09 | 筋萎縮性側索硬化症(als)を処置する方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US11859179B2 (https=) |
| EP (1) | EP3622073A4 (https=) |
| JP (1) | JP7327803B2 (https=) |
| AU (1) | AU2018264996A1 (https=) |
| CA (1) | CA3059213A1 (https=) |
| WO (1) | WO2018208972A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2942515C (en) | 2014-03-18 | 2025-12-09 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| AU2016270587B2 (en) | 2015-05-29 | 2020-12-24 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a disruption in a C9ORF72 locus |
| CN109862785B (zh) | 2016-09-30 | 2022-09-06 | 瑞泽恩制药公司 | C9orf72基因座中具有六核苷酸重复扩增的非人类动物 |
| US11859179B2 (en) | 2017-05-09 | 2024-01-02 | University Of Massachusetts | Methods of treating amyotrophic lateral sclerosis (ALS) |
| CN111448321A (zh) | 2017-09-22 | 2020-07-24 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
| KR102925054B1 (ko) | 2018-12-20 | 2026-02-10 | 리제너론 파마슈티칼스 인코포레이티드 | 뉴클레아제-매개 반복부 팽창 |
| TW202521561A (zh) * | 2019-04-23 | 2025-06-01 | 美商聖加莫治療股份有限公司 | 染色體9開放讀框72基因表現之調節子及其用途 |
| AU2021237633A1 (en) * | 2020-03-18 | 2022-10-06 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of C9orf72 |
| EP4222264A1 (en) * | 2020-09-30 | 2023-08-09 | CRISPR Therapeutics AG | Materials and methods for treatment of amyotrophic lateral sclerosis |
| GB202105455D0 (en) * | 2021-04-16 | 2021-06-02 | Ucl Business Ltd | Composition |
| WO2023025103A1 (en) * | 2021-08-22 | 2023-03-02 | Huigene Therapeutics Co., Ltd. | Crispr-cas13 system for treating sod1-associated diseases |
| EP4430188A1 (en) * | 2021-11-09 | 2024-09-18 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of amyotrophic lateral sclerosis |
Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015057727A1 (en) | 2013-10-14 | 2015-04-23 | Isis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| WO2016112132A1 (en) | 2015-01-06 | 2016-07-14 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| WO2016210372A2 (en) | 2015-06-25 | 2016-12-29 | University Of Southern California | Methods to treat neurological diseases |
| WO2017109757A1 (en) | 2015-12-23 | 2017-06-29 | Crispr Therapeutics Ag | Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration |
| WO2018064600A1 (en) | 2016-09-30 | 2018-04-05 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus |
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| JP2020519284A (ja) | 2020-07-02 |
| US20240167025A1 (en) | 2024-05-23 |
| EP3622073A4 (en) | 2021-01-06 |
| US11859179B2 (en) | 2024-01-02 |
| US12529051B2 (en) | 2026-01-20 |
| WO2018208972A1 (en) | 2018-11-15 |
| CA3059213A1 (en) | 2018-11-15 |
| AU2018264996A1 (en) | 2019-12-05 |
| EP3622073A1 (en) | 2020-03-18 |
| US20200354716A1 (en) | 2020-11-12 |
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