JP2025501755A5 - - Google Patents
Info
- Publication number
- JP2025501755A5 JP2025501755A5 JP2024538030A JP2024538030A JP2025501755A5 JP 2025501755 A5 JP2025501755 A5 JP 2025501755A5 JP 2024538030 A JP2024538030 A JP 2024538030A JP 2024538030 A JP2024538030 A JP 2024538030A JP 2025501755 A5 JP2025501755 A5 JP 2025501755A5
- Authority
- JP
- Japan
- Prior art keywords
- aso
- restricted
- modification
- use according
- nucleotides
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163265989P | 2021-12-23 | 2021-12-23 | |
| US63/265,989 | 2021-12-23 | ||
| PCT/US2022/082380 WO2023122800A1 (en) | 2021-12-23 | 2022-12-23 | Therapeutic treatment for fragile x-associated disorder |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025501755A JP2025501755A (ja) | 2025-01-23 |
| JP2025501755A5 true JP2025501755A5 (https=) | 2026-01-06 |
Family
ID=85199427
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024538030A Pending JP2025501755A (ja) | 2021-12-23 | 2022-12-23 | 脆弱ｘ関連障害に対する治療的処置 |
Country Status (9)
| Country | Link |
|---|---|
| EP (1) | EP4453212A1 (https=) |
| JP (1) | JP2025501755A (https=) |
| KR (1) | KR20240133725A (https=) |
| CN (1) | CN118679257A (https=) |
| AU (1) | AU2022417615A1 (https=) |
| CA (1) | CA3240593A1 (https=) |
| IL (1) | IL313726A (https=) |
| MX (1) | MX2024007908A (https=) |
| WO (1) | WO2023122800A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025240947A1 (en) * | 2024-05-17 | 2025-11-20 | University Of Massachusetts | Therapeutic treatment for fragile x-associated disorder |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3854480A (en) | 1969-04-01 | 1974-12-17 | Alza Corp | Drug-delivery system |
| US4452775A (en) | 1982-12-03 | 1984-06-05 | Syntex (U.S.A.) Inc. | Cholesterol matrix delivery system for sustained release of macromolecules |
| US5039660A (en) | 1988-03-02 | 1991-08-13 | Endocon, Inc. | Partially fused peptide pellet |
| WO1998010090A1 (en) | 1996-09-06 | 1998-03-12 | St. Elizabeth's Medical Center Of Boston, Inc. | Gata-6 transcription factor: compositions and methods |
| EP2595663A4 (en) * | 2010-07-19 | 2014-03-05 | Isis Pharmaceuticals Inc | MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION |
| EP2723902A4 (en) * | 2011-06-24 | 2015-02-18 | Murdoch Childrens Res Inst | TREATMENT AND DIAGNOSIS OF EPIGENETIC DISORDERS AND DISEASES |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| AU2013335451C1 (en) | 2012-10-23 | 2024-07-04 | Toolgen Incorporated | Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| CN113355357B (zh) | 2012-12-12 | 2024-12-03 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| EP2840140B2 (en) | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| IL239344B2 (en) | 2012-12-12 | 2024-06-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| CA2921457A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Heterochromatin forming non-coding rnas |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| WO2018170290A1 (en) * | 2017-03-15 | 2018-09-20 | Fulcrum Therapeutics, Inc. | Compositions and methods for increasing fmr1 expression |
| GB2574243A (en) * | 2018-05-31 | 2019-12-04 | The Govening Council Of The Univ Of Toronto | Adeno-associated viral vector-mediated gene therapy for treating fragile X-associated disorders |
| CA3130854A1 (en) * | 2019-02-26 | 2020-09-03 | Nogra Pharma Limited | Fragile x mental retardation protein interfering oligonucleotides and methods of using same |
| CN120022385A (zh) * | 2019-05-07 | 2025-05-23 | 洛桑联邦理工学院 | Fmrp及癌症治疗 |
| CN113151406B (zh) * | 2021-03-25 | 2022-06-21 | 深圳会众生物技术有限公司 | Fmr1基因cgg重复数及甲基化检测试剂盒和检测方法 |
-
2022
- 2022-12-23 JP JP2024538030A patent/JP2025501755A/ja active Pending
- 2022-12-23 CA CA3240593A patent/CA3240593A1/en active Pending
- 2022-12-23 IL IL313726A patent/IL313726A/en unknown
- 2022-12-23 MX MX2024007908A patent/MX2024007908A/es unknown
- 2022-12-23 KR KR1020247024797A patent/KR20240133725A/ko active Pending
- 2022-12-23 WO PCT/US2022/082380 patent/WO2023122800A1/en not_active Ceased
- 2022-12-23 AU AU2022417615A patent/AU2022417615A1/en active Pending
- 2022-12-23 EP EP22854728.7A patent/EP4453212A1/en active Pending
- 2022-12-23 CN CN202280089598.9A patent/CN118679257A/zh active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7442574B2 (ja) | Ｌｐａの遺伝子発現の阻害のための組成物及び方法 | |
| AU2004221760B2 (en) | Short interfering RNA (siRNA) analogues | |
| EP1937312B1 (en) | Chimeric oligomeric compounds for modulation of splicing | |
| EP3484524B1 (en) | Compounds and methods for modulation of smn2 | |
| WO2020165077A1 (en) | Antisense oligonucleotides for nucleic acid editing | |
| JP2021533804A5 (https=) | ||
| JP2016528897A (ja) | Ｒｎａを調節するための組成物および方法 | |
| JP2022545118A5 (https=) | ||
| TW201639962A (zh) | Ｒｎａ干擾劑 | |
| TW201702378A (zh) | 反義誘導之酸性α－葡萄苷酶中的外顯子２包含 | |
| CA2457528A1 (en) | Rna interference mediated inhibition of hepatitis c virus (hcv) gene expression using short interfering nucleic acid (sina) | |
| CA2921457A1 (en) | Heterochromatin forming non-coding rnas | |
| CA3118167A1 (en) | Bispecific antisense oligonucleotides for dystrophin exon skipping | |
| JP2024516183A5 (https=) | ||
| RU2020125769A (ru) | Олигонуклеотиды для модуляции экспрессии tmem106b | |
| JP2025501755A5 (https=) | ||
| EP4396352A2 (en) | Compounds and methods for reducing dmpk expression | |
| US20260062703A1 (en) | Delivery of RNA Therapeutics Using Circular Prodrug Nucleic Acids | |
| TW202329983A (zh) | Ｒｎａ編輯 | |
| JP2024536132A5 (https=) | ||
| JP2026002846A (ja) | Ａｎｇｐｔｌ２アンチセンスオリゴヌクレオチドおよびその使用 | |
| CN114867856A (zh) | Saraf抑制剂用于治疗乙型肝炎病毒感染的用途 | |
| US6238917B1 (en) | Asymmetric hammerhead ribozymes | |
| EP4332221A1 (en) | Threose nucleic acid antisense oligonucleotides and methods thereof | |
| CN112218950A (zh) | 心肌损伤治疗药 |