JP2022529424A5 - - Google Patents
Info
- Publication number
- JP2022529424A5 JP2022529424A5 JP2021560850A JP2021560850A JP2022529424A5 JP 2022529424 A5 JP2022529424 A5 JP 2022529424A5 JP 2021560850 A JP2021560850 A JP 2021560850A JP 2021560850 A JP2021560850 A JP 2021560850A JP 2022529424 A5 JP2022529424 A5 JP 2022529424A5
- Authority
- JP
- Japan
- Prior art keywords
- dystrophin gene
- sequence
- vector
- exon
- seq
- 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.)
- Granted
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962833759P | 2019-04-14 | 2019-04-14 | |
| US62/833,759 | 2019-04-14 | ||
| PCT/US2020/028154 WO2020214613A1 (en) | 2019-04-14 | 2020-04-14 | Crispr/cas-based genome editing composition for restoring dystrophin function |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022529424A JP2022529424A (ja) | 2022-06-22 |
| JP2022529424A5 true JP2022529424A5 (https=) | 2023-04-20 |
| JP7827288B2 JP7827288B2 (ja) | 2026-03-10 |
Family
ID=72837592
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021560850A Active JP7827288B2 (ja) | 2019-04-14 | 2020-04-14 | ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにしたゲノム編集組成物 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20220195406A1 (https=) |
| EP (1) | EP3930766A4 (https=) |
| JP (1) | JP7827288B2 (https=) |
| AR (1) | AR118668A1 (https=) |
| TW (1) | TW202100748A (https=) |
| WO (1) | WO2020214613A1 (https=) |
Families Citing this family (17)
| 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 |
| US11028388B2 (en) | 2014-03-05 | 2021-06-08 | Editas Medicine, Inc. | CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| WO2017035416A2 (en) | 2015-08-25 | 2017-03-02 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
| 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 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| 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 |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| US20230272428A1 (en) * | 2019-12-16 | 2023-08-31 | President And Fellows Of Harvard College | Methods and compositions for correction of dmd mutations |
| US20230257723A1 (en) * | 2020-04-27 | 2023-08-17 | Duke University | Crispr/cas9 therapies for correcting duchenne muscular dystrophy by targeted genomic integration |
| AU2021345112A1 (en) * | 2020-09-15 | 2023-04-27 | Research Institute At Nationwide Children's Hospital | Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy |
| WO2022221741A1 (en) * | 2021-04-16 | 2022-10-20 | Editas Medicine, Inc. | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
| IL317874A (en) | 2022-06-24 | 2025-02-01 | Tune Therapeutics Inc | Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression |
| CN115820642B (zh) * | 2022-11-11 | 2023-10-10 | 昆明理工大学 | 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| WO2016187717A1 (en) * | 2015-05-26 | 2016-12-01 | Exerkine Corporation | Exosomes useful for genome editing |
| AU2016344609B2 (en) * | 2015-10-28 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of duchenne muscular dystrophy |
| US20190127713A1 (en) * | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP4321623A3 (en) * | 2016-07-15 | 2024-05-15 | Salk Institute for Biological Studies | Methods and compositions for genome editing in non-dividing cells |
| AU2019216321A1 (en) * | 2018-01-31 | 2020-07-30 | The Board Of Regents Of The University Of Texas System | Compositions and methods for correcting dystrophin mutations in human cardiomyocytes |
-
2020
- 2020-04-14 AR ARP200101046A patent/AR118668A1/es unknown
- 2020-04-14 EP EP20791785.7A patent/EP3930766A4/en active Pending
- 2020-04-14 JP JP2021560850A patent/JP7827288B2/ja active Active
- 2020-04-14 US US17/603,329 patent/US20220195406A1/en active Pending
- 2020-04-14 WO PCT/US2020/028154 patent/WO2020214613A1/en not_active Ceased
- 2020-04-14 TW TW109112554A patent/TW202100748A/zh unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022529424A5 (https=) | ||
| JP2022529631A5 (https=) | ||
| JP2020500541A5 (https=) | ||
| JP2024009857A5 (https=) | ||
| JP2020519284A5 (https=) | ||
| JP2018516978A5 (https=) | ||
| JP2021516046A5 (https=) | ||
| JP2020019772A5 (https=) | ||
| JP2023515692A5 (https=) | ||
| JP2019525759A5 (https=) | ||
| JP2024099770A5 (https=) | ||
| JP2020522269A5 (https=) | ||
| RU2017143997A (ru) | Генное редактирование глубоких интронных мутаций | |
| JP2021511803A5 (https=) | ||
| JP2017509632A5 (https=) | ||
| US11814642B2 (en) | Manufacturing and use of recombinant AAV vectors | |
| JP2017535266A5 (https=) | ||
| JP2021523702A5 (https=) | ||
| JP2022551744A5 (https=) | ||
| CA3158518A1 (en) | Triple function adeno-associated virus (aav)vectors for the treatment of c9orf72 associated diseases | |
| JP2022524434A5 (https=) | ||
| JP2025029049A5 (https=) | ||
| JP2023551873A5 (https=) | ||
| US20220380798A1 (en) | AAV Expression Cassette and AAV Vectors Comprising the Same | |
| JP2023059858A5 (https=) |