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 (enExample) | 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 (enExample) |
| EP (1) | EP3930766A4 (enExample) |
| JP (1) | JP7827288B2 (enExample) |
| AR (1) | AR118668A1 (enExample) |
| TW (1) | TW202100748A (enExample) |
| WO (1) | WO2020214613A1 (enExample) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | 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 |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| KR20240132120A (ko) | 2015-08-25 | 2024-09-02 | 듀크 유니버시티 | Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법 |
| WO2017066497A2 (en) | 2015-10-13 | 2017-04-20 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| US12214054B2 (en) | 2015-11-30 | 2025-02-04 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| EP3443081A4 (en) | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD 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 |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| TWI887234B (zh) | 2019-04-14 | 2025-06-21 | 美國公爵大學 | 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除 |
| JP2023507174A (ja) * | 2019-12-16 | 2023-02-21 | プレジデント アンド フェローズ オブ ハーバード カレッジ | Ｄｍｄ変異の修正のための方法及び組成物 |
| JP2023522788A (ja) * | 2020-04-27 | 2023-05-31 | デューク ユニバーシティ | 標的化されたゲノム組込みによってデュシェンヌ型筋ジストロフィーを矯正するためのｃｒｉｓｐｒ／ｃａｓ９療法 |
| JP2023541444A (ja) * | 2020-09-15 | 2023-10-02 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィー患者における多様なｄｍｄ変異の補正のためのａａｖ媒介性の相同性非依存的標的化組み込み遺伝子編集 |
| 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) |
| WO2022251181A1 (en) * | 2021-05-25 | 2022-12-01 | The Board Of Regents Of The University Of Texas System | Correction of duchenne muscular dystrophy mutations with all-in-one adeno-associated virus-delivered single-cut crispr |
| CA3260237A1 (en) | 2022-06-24 | 2023-12-28 | Tune Therapeutics, Inc. | Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression |
| 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 |
| EP4279084B1 (en) | 2015-10-28 | 2025-06-11 | Vertex Pharmaceuticals Inc. | Materials and methods for treatment of duchenne muscular dystrophy |
| EP3443081A4 (en) * | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE |
| CA3030587A1 (en) | 2016-07-15 | 2018-01-18 | Salk Institute For Biological Studies | Methods and compositions for genome editing in non-dividing cells |
| EP3746557A1 (en) * | 2018-01-31 | 2020-12-09 | The Board Of Regents Of The University Of Texas System | Compositions and methods for correcting dystrophin mutations in human cardiomyocytes |
-
2020
- 2020-04-14 TW TW109112554A patent/TW202100748A/zh unknown
- 2020-04-14 US US17/603,329 patent/US20220195406A1/en active Pending
- 2020-04-14 JP JP2021560850A patent/JP7827288B2/ja active Active
- 2020-04-14 AR ARP200101046A patent/AR118668A1/es unknown
- 2020-04-14 EP EP20791785.7A patent/EP3930766A4/en active Pending
- 2020-04-14 WO PCT/US2020/028154 patent/WO2020214613A1/en not_active Ceased
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