JP2021500041A5 - - Google Patents
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- JP2021500041A5 JP2021500041A5 JP2020522905A JP2020522905A JP2021500041A5 JP 2021500041 A5 JP2021500041 A5 JP 2021500041A5 JP 2020522905 A JP2020522905 A JP 2020522905A JP 2020522905 A JP2020522905 A JP 2020522905A JP 2021500041 A5 JP2021500041 A5 JP 2021500041A5
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- grch38
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- target protein
- chr2
- chr1
- Prior art date
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024015750A JP7783920B2 (ja) | 2017-10-23 | 2024-02-05 | ナンセンス変異依存ｒｎａ分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762575924P | 2017-10-23 | 2017-10-23 | |
| US62/575,924 | 2017-10-23 | ||
| US201862667200P | 2018-05-04 | 2018-05-04 | |
| US62/667,200 | 2018-05-04 | ||
| PCT/US2018/057165 WO2019084050A1 (en) | 2017-10-23 | 2018-10-23 | ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DECLINE OF NON-SENSE MEDIATED MRNA |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024015750A Division JP7783920B2 (ja) | 2017-10-23 | 2024-02-05 | ナンセンス変異依存ｒｎａ分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021500041A JP2021500041A (ja) | 2021-01-07 |
| JP2021500041A5 true JP2021500041A5 (enExample) | 2021-12-02 |
| JP7475272B2 JP7475272B2 (ja) | 2024-04-26 |
Family
ID=66247689
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020522905A Active JP7475272B2 (ja) | 2017-10-23 | 2018-10-23 | ナンセンス変異依存ｒｎａ分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
| JP2024015750A Active JP7783920B2 (ja) | 2017-10-23 | 2024-02-05 | ナンセンス変異依存ｒｎａ分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024015750A Active JP7783920B2 (ja) | 2017-10-23 | 2024-02-05 | ナンセンス変異依存ｒｎａ分解機構に基づく状態および疾患の処置のためのアンチセンスオリゴマー |
Country Status (23)
| Country | Link |
|---|---|
| US (2) | US20210268667A1 (enExample) |
| EP (2) | EP3700570B1 (enExample) |
| JP (2) | JP7475272B2 (enExample) |
| KR (2) | KR20250025519A (enExample) |
| CN (2) | CN120290560A (enExample) |
| AU (1) | AU2018355237B2 (enExample) |
| BR (1) | BR112020007881A2 (enExample) |
| CA (1) | CA3079729A1 (enExample) |
| DK (1) | DK3700570T3 (enExample) |
| ES (1) | ES3015767T3 (enExample) |
| FI (1) | FI3700570T3 (enExample) |
| GB (2) | GB2610100B (enExample) |
| HR (1) | HRP20250322T1 (enExample) |
| HU (1) | HUE070436T2 (enExample) |
| IL (1) | IL274023A (enExample) |
| LT (1) | LT3700570T (enExample) |
| PL (1) | PL3700570T3 (enExample) |
| PT (1) | PT3700570T (enExample) |
| RS (1) | RS66633B1 (enExample) |
| SG (1) | SG11202003692YA (enExample) |
| SI (1) | SI3700570T1 (enExample) |
| SM (1) | SMT202500127T1 (enExample) |
| WO (1) | WO2019084050A1 (enExample) |
Families Citing this family (60)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| WO2016040748A1 (en) | 2014-09-12 | 2016-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for detection of smn protein in a subject and treatment of a subject |
| EP3201339A4 (en) | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| KR102422625B1 (ko) | 2015-10-09 | 2022-07-20 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| WO2017066497A2 (en) | 2015-10-13 | 2017-04-20 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| WO2017095967A2 (en) | 2015-11-30 | 2017-06-08 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| US11198867B2 (en) | 2016-06-16 | 2021-12-14 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of SMN expression |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| SG11202001590RA (en) | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
| EP3788169A4 (en) | 2018-05-04 | 2022-08-10 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| CN109593771B (zh) * | 2018-07-27 | 2022-03-29 | 四川大学华西医院 | 一种人类map2k5第1100位碱基突变基因及其检测试剂盒 |
| WO2020191212A1 (en) | 2019-03-20 | 2020-09-24 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| TWI874376B (zh) | 2019-03-29 | 2025-03-01 | 美商Ionis製藥公司 | 用於調節ube3a-ats 之化合物及方法 |
| BR112022002905A2 (pt) * | 2019-08-19 | 2022-07-12 | Stoke Therapeutics Inc | Composições e métodos para modular splicing e expressão de proteína |
| US11618900B2 (en) * | 2019-11-01 | 2023-04-04 | The Johns Hopkins University | Modulating SYNGAP |
| CA3159162A1 (en) * | 2019-12-06 | 2021-06-10 | Isabel AZNAREZ | Antisense oligomers for treatment of conditions and diseases |
| BR112022016238A2 (pt) | 2020-02-28 | 2022-10-11 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular smn2 |
| WO2021178769A1 (en) * | 2020-03-06 | 2021-09-10 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating kcnq2 |
| WO2021222328A1 (en) * | 2020-04-27 | 2021-11-04 | Duke University | Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1) |
| MX2022014151A (es) * | 2020-05-11 | 2022-11-30 | Stoke Therapeutics Inc | Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades. |
| IL298070A (en) * | 2020-05-11 | 2023-01-01 | The Florey Inst Of Neuroscience And Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in syngap1 |
| CA3186629A1 (en) * | 2020-07-22 | 2022-01-27 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in scn2a |
| CA3186935A1 (en) | 2020-08-07 | 2022-02-10 | Paymaan JAFAR-NEJAD | Compounds and methods for modulating scn2a |
| CA3193706A1 (en) * | 2020-10-02 | 2022-04-07 | PYC Therapeutics Limited | Treatment of optic atrophy |
| US20240018522A1 (en) * | 2020-10-26 | 2024-01-18 | Remix Therapeutics Inc. | Oligonucleotides useful for modulation of splicing |
| CN117413061A (zh) * | 2021-02-03 | 2024-01-16 | 斯托克制药公司 | 用于治疗与多囊蛋白表达相关的疾患和疾病的组合物 |
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