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) | EP4494706A3 (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) | GB2610100B (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 |
|---|---|---|---|---|
| EP3597741A1 (en) | 2012-04-27 | 2020-01-22 | 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 |
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| EP3359685B1 (en) | 2015-10-09 | 2026-01-28 | University Of Southampton | Modulation of gene expression for deregulated protein expression |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| EA201891317A3 (ru) | 2015-11-30 | 2019-04-30 | Дьюк Юниверсити | Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения |
| EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
| 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 |
| WO2017218884A1 (en) | 2016-06-16 | 2017-12-21 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of smn expression |
| WO2018017754A1 (en) | 2016-07-19 | 2018-01-25 | 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 |
| WO2019144061A1 (en) | 2018-01-19 | 2019-07-25 | Duke University | Genome engineering with crispr-cas systems in eukaryotes |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | 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 |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| US12595479B2 (en) | 2019-03-20 | 2026-04-07 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| FI3947684T3 (fi) | 2019-03-29 | 2025-05-27 | Ionis Pharmaceuticals Inc | Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi |
| 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 |
| CR20220485A (es) | 2020-02-28 | 2022-11-10 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular smn2 |
| US20230124616A1 (en) * | 2020-03-06 | 2023-04-20 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating kcnq2 |
| EP4125350A4 (en) * | 2020-04-27 | 2024-04-03 | Duke University | Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1) |
| BR112022022889A2 (pt) * | 2020-05-11 | 2023-04-04 | Stoke Therapeutics Inc | Oligômeros antissentido de opa1 para tratamento de condições e doenças |
| AU2021272832A1 (en) * | 2020-05-11 | 2022-12-15 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in SYNGAP1 |
| CN116368227A (zh) * | 2020-07-22 | 2023-06-30 | 弗洛里神经科学与心理健康研究所 | 用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法 |
| IL300258A (en) * | 2020-08-07 | 2023-03-01 | Ionis Pharmaceuticals Inc | Compounds and methods for modulating SCN2A |
| BR112023005748A2 (pt) * | 2020-10-02 | 2023-05-09 | Pyc Therapeutics Ltd | Tratamento de atrofia óptica |
| US20240018522A1 (en) * | 2020-10-26 | 2024-01-18 | Remix Therapeutics Inc. | Oligonucleotides useful for modulation of splicing |
| AU2022215577A1 (en) * | 2021-02-03 | 2023-09-21 | Stoke Therapeutics, Inc. | Compositions for treatment of conditions and diseases associated with polycystin expression |
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