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
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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 (https=) | 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 (https=) |
| EP (2) | EP3700570B1 (https=) |
| JP (2) | JP7475272B2 (https=) |
| KR (2) | KR102774070B1 (https=) |
| CN (2) | CN111936163B (https=) |
| AU (2) | AU2018355237B2 (https=) |
| BR (1) | BR112020007881A2 (https=) |
| CA (1) | CA3079729A1 (https=) |
| DK (1) | DK3700570T3 (https=) |
| ES (1) | ES3015767T3 (https=) |
| FI (1) | FI3700570T3 (https=) |
| GB (2) | GB2584204B (https=) |
| HR (1) | HRP20250322T1 (https=) |
| HU (1) | HUE070436T2 (https=) |
| IL (1) | IL274023A (https=) |
| LT (1) | LT3700570T (https=) |
| PL (1) | PL3700570T3 (https=) |
| PT (1) | PT3700570T (https=) |
| RS (1) | RS66633B1 (https=) |
| SG (1) | SG11202003692YA (https=) |
| SI (1) | SI3700570T1 (https=) |
| SM (1) | SMT202500127T1 (https=) |
| WO (1) | WO2019084050A1 (https=) |
Families Citing this family (64)
| 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 |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| 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 |
| JP7490211B2 (ja) | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Ｃｐｆ１に基づくゲノム編集の治療適用 |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | 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 |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| US12595479B2 (en) | 2019-03-20 | 2026-04-07 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| PT3947684T (pt) | 2019-03-29 | 2025-05-19 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular ube3a‑ats |
| EP4017979A4 (en) * | 2019-08-19 | 2024-03-27 | Stoke Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION |
| US11618900B2 (en) * | 2019-11-01 | 2023-04-04 | The Johns Hopkins University | Modulating SYNGAP |
| BR112022010882A2 (pt) * | 2019-12-06 | 2022-10-04 | Stoke Therapeutics Inc | Oligômeros antissenso para tratamento de condições e doenças |
| CN120505310A (zh) * | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
| 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) |
| CN115916977A (zh) * | 2020-05-11 | 2023-04-04 | 弗洛里神经科学与心理健康研究所 | 用于治疗与syngap1中功能失去突变相关的障碍的组合物和方法 |
| CN115867657A (zh) * | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| KR20230095056A (ko) * | 2020-07-22 | 2023-06-28 | 더 플로레이 인스티튜트 오브 뉴로사이언스 앤드 멘탈 헬스 | Scn2a에서 기능-상실 돌연변이와 연관된 장애를 치료하기 위한 조성물 및 방법 |
| IL300258A (en) * | 2020-08-07 | 2023-03-01 | Ionis Pharmaceuticals Inc | Compounds and methods for modulating SCN2A |
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