JP2022529631A5 - - Google Patents
Info
- Publication number
- JP2022529631A5 JP2022529631A5 JP2021560849A JP2021560849A JP2022529631A5 JP 2022529631 A5 JP2022529631 A5 JP 2022529631A5 JP 2021560849 A JP2021560849 A JP 2021560849A JP 2021560849 A JP2021560849 A JP 2021560849A JP 2022529631 A5 JP2022529631 A5 JP 2022529631A5
- Authority
- JP
- Japan
- Prior art keywords
- grna
- vector
- promoter
- cas9 protein
- cas9
- 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.)
- Pending
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025014814A JP2025087680A (ja) | 2019-04-14 | 2025-01-31 | デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのａａｖベクター媒介欠失 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962833760P | 2019-04-14 | 2019-04-14 | |
| US62/833,760 | 2019-04-14 | ||
| PCT/US2020/028148 WO2020214609A1 (en) | 2019-04-14 | 2020-04-14 | Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025014814A Division JP2025087680A (ja) | 2019-04-14 | 2025-01-31 | デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのａａｖベクター媒介欠失 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022529631A JP2022529631A (ja) | 2022-06-23 |
| JP2022529631A5 true JP2022529631A5 (https=) | 2023-04-21 |
Family
ID=72837591
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021560849A Pending JP2022529631A (ja) | 2019-04-14 | 2020-04-14 | デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのａａｖベクター媒介欠失 |
| JP2025014814A Pending JP2025087680A (ja) | 2019-04-14 | 2025-01-31 | デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのａａｖベクター媒介欠失 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025014814A Pending JP2025087680A (ja) | 2019-04-14 | 2025-01-31 | デュシェンヌ型筋ジストロフィーの処置のための大きい突然変異ホットスポットのａａｖベクター媒介欠失 |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US20220184229A1 (https=) |
| EP (1) | EP3931331A4 (https=) |
| JP (2) | JP2022529631A (https=) |
| KR (1) | KR20210151916A (https=) |
| CN (1) | CN113785063A (https=) |
| AR (1) | AR118670A1 (https=) |
| AU (1) | AU2020257898B2 (https=) |
| BR (1) | BR112021020515A2 (https=) |
| CA (1) | CA3137248A1 (https=) |
| CL (1) | CL2021002680A1 (https=) |
| CO (1) | CO2021013692A2 (https=) |
| EA (1) | EA202192815A1 (https=) |
| IL (1) | IL287163A (https=) |
| MX (1) | MX2021012511A (https=) |
| SG (1) | SG11202111172QA (https=) |
| TW (2) | TWI887234B (https=) |
| WO (1) | WO2020214609A1 (https=) |
Families Citing this family (16)
| 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 |
| 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 |
| US20230346978A1 (en) * | 2020-04-14 | 2023-11-02 | University Of Massachusetts | Dcas13-mediated therapeutic rna base editing for in vivo gene therapy |
| WO2022087321A1 (en) * | 2020-10-21 | 2022-04-28 | Duke University | Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy |
| MX2024002988A (es) * | 2021-09-08 | 2024-06-11 | Flagship Pioneering Innovations Vi Llc | Composiciones y metodos moduladores de la serpina. |
| IL317874A (en) | 2022-06-24 | 2025-02-01 | Tune Therapeutics Inc | Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression |
| WO2024193704A1 (en) * | 2023-03-22 | 2024-09-26 | Huidagene Therapeutics Co., Ltd. | Guide nucleic acids targeting dmd and uses thereof |
| WO2025221969A2 (en) | 2024-04-18 | 2025-10-23 | Sarepta Therapeutics, Inc. | Genetic constructs for gene editing |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2005035718A2 (en) * | 2003-10-03 | 2005-04-21 | Welgen, Inc. | Biderectional promoters for small rna expression |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| ES2788426T3 (es) * | 2014-06-16 | 2020-10-21 | Univ Johns Hopkins | Composiciones y métodos para la expresión de ARNs de guía de CRISPR utilizando el promotor de H1 |
| AU2016344609B2 (en) * | 2015-10-28 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of duchenne muscular dystrophy |
| KR102787119B1 (ko) * | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| EP3199632A1 (en) * | 2016-01-26 | 2017-08-02 | ACIB GmbH | Temperature-inducible crispr/cas system |
| WO2017139505A2 (en) * | 2016-02-11 | 2017-08-17 | The Regents Of The University Of California | Methods and compositions for modifying a mutant dystrophin gene in a cell's genome |
| US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
| US10687520B2 (en) * | 2017-03-07 | 2020-06-23 | The Board Of Regents Of The University Of Texas System | Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44 |
| US11779653B2 (en) * | 2017-09-29 | 2023-10-10 | The Regents Of The University Of California | Multi-armed polyrotaxane platform for protected nucleic acid delivery |
-
2020
- 2020-04-14 WO PCT/US2020/028148 patent/WO2020214609A1/en not_active Ceased
- 2020-04-14 MX MX2021012511A patent/MX2021012511A/es unknown
- 2020-04-14 CA CA3137248A patent/CA3137248A1/en active Pending
- 2020-04-14 US US17/603,330 patent/US20220184229A1/en active Pending
- 2020-04-14 AR ARP200101048A patent/AR118670A1/es unknown
- 2020-04-14 EP EP20790851.8A patent/EP3931331A4/en active Pending
- 2020-04-14 AU AU2020257898A patent/AU2020257898B2/en active Active
- 2020-04-14 SG SG11202111172QA patent/SG11202111172QA/en unknown
- 2020-04-14 CN CN202080028248.2A patent/CN113785063A/zh active Pending
- 2020-04-14 TW TW109112555A patent/TWI887234B/zh active
- 2020-04-14 TW TW114119381A patent/TW202536186A/zh unknown
- 2020-04-14 BR BR112021020515A patent/BR112021020515A2/pt unknown
- 2020-04-14 JP JP2021560849A patent/JP2022529631A/ja active Pending
- 2020-04-14 KR KR1020217036783A patent/KR20210151916A/ko active Pending
- 2020-04-14 EA EA202192815A patent/EA202192815A1/ru unknown
-
2021
- 2021-10-11 IL IL287163A patent/IL287163A/en unknown
- 2021-10-13 CL CL2021002680A patent/CL2021002680A1/es unknown
- 2021-10-14 CO CONC2021/0013692A patent/CO2021013692A2/es unknown
-
2025
- 2025-01-31 JP JP2025014814A patent/JP2025087680A/ja active Pending