JP2022115976A5 - - Google Patents
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- JP2022115976A5 JP2022115976A5 JP2022076261A JP2022076261A JP2022115976A5 JP 2022115976 A5 JP2022115976 A5 JP 2022115976A5 JP 2022076261 A JP2022076261 A JP 2022076261A JP 2022076261 A JP2022076261 A JP 2022076261A JP 2022115976 A5 JP2022115976 A5 JP 2022115976A5
- Authority
- JP
- Japan
- Prior art keywords
- base sequence
- sequence encoding
- region
- nuclease
- polynucleotide
- 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
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Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862768474P | 2018-11-16 | 2018-11-16 | |
| US62/768,474 | 2018-11-16 | ||
| US201962861039P | 2019-06-13 | 2019-06-13 | |
| US62/861,039 | 2019-06-13 | ||
| US201962931925P | 2019-11-07 | 2019-11-07 | |
| US62/931,925 | 2019-11-07 | ||
| PCT/JP2019/045716 WO2020101042A1 (en) | 2018-11-16 | 2019-11-15 | Method for treating muscular dystrophy by targeting utrophin gene |
| JP2021551108A JP7069426B2 (ja) | 2018-11-16 | 2019-11-15 | ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021551108A Division JP7069426B2 (ja) | 2018-11-16 | 2019-11-15 | ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022115976A JP2022115976A (ja) | 2022-08-09 |
| JP2022115976A5 true JP2022115976A5 (cg-RX-API-DMAC7.html) | 2022-11-24 |
Family
ID=68988228
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021551108A Active JP7069426B2 (ja) | 2018-11-16 | 2019-11-15 | ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 |
| JP2022076261A Pending JP2022115976A (ja) | 2018-11-16 | 2022-05-02 | ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021551108A Active JP7069426B2 (ja) | 2018-11-16 | 2019-11-15 | ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 |
Country Status (15)
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102008496B1 (ko) * | 2013-03-25 | 2019-08-07 | 삼성전자주식회사 | 단일 센서를 이용하여 기구물의 이동 방향 및 속도를 검출하는 방법 및 장치. |
| 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 |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| AU2019255789B2 (en) | 2018-04-19 | 2025-11-20 | The Regents Of The University Of California | Compositions and methods for gene editing |
| JP7412001B2 (ja) * | 2018-06-08 | 2024-01-12 | 株式会社モダリス | 改変されたＣａｓ９タンパク質及びその用途 |
| AU2019317066A1 (en) * | 2018-08-07 | 2021-02-18 | Modalis Therapeutics Corporation | Novel transcription activator |
| 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 |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| EP4222263A1 (en) | 2020-09-29 | 2023-08-09 | Genethon | Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements and therapeutic use thereof |
| US20240050406A1 (en) * | 2021-04-26 | 2024-02-15 | Ksb Tugen Inc. | Composition comprising oxiracetam for preventing or treating muscular disease |
| KR102421411B1 (ko) | 2021-10-29 | 2022-07-18 | 주식회사 케이에스비튜젠 | 옥시라세탐을 포함하는 근이영양증 예방 또는 치료용 조성물 |
| CN113563428B (zh) * | 2021-06-22 | 2022-08-02 | 呈诺再生医学科技(珠海横琴新区)有限公司 | 特定空间结构多肽及其在制备iPSC中的应用 |
| US20230279442A1 (en) * | 2021-12-15 | 2023-09-07 | Versitech Limited | Engineered cas9-nucleases and method of use thereof |
| WO2023190935A1 (en) * | 2022-03-31 | 2023-10-05 | Modalis Therapeutics Corporation | Method for treating myopathies by targeting titin gene |
| CN116218849B (zh) * | 2023-01-18 | 2024-01-23 | 昆明理工大学 | 针对utrophin激活治疗杜氏肌营养不良的sgRNA和应用 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2001083695A2 (en) * | 2000-04-28 | 2001-11-08 | Xiao Xiao | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| EP1390490B1 (en) | 2001-05-24 | 2009-04-15 | Genzyme Corporation | Muscle-specific expression vectors |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| US7557197B2 (en) | 2004-01-28 | 2009-07-07 | Oregon Health & Science University | Human soluble neuropilin-1 primary polyadenylation signal and uses thereof |
| ITRM20070523A1 (it) | 2007-10-05 | 2009-04-06 | Consiglio Nazionale Ricerche | Acido nucleico codificante per una proteina regolatrice specifica della trascrizione dell'utrofina proteina da esso codificata e sue applicazioni |
| US10738326B2 (en) | 2010-10-27 | 2020-08-11 | Jichi Medical University | Adeno-associated virus vector for gene transfer to nervous system cells |
| CN104540946A (zh) * | 2012-05-16 | 2015-04-22 | Rana医疗有限公司 | 用于调节utrn表达的组合物和方法 |
| ES2728782T3 (es) | 2012-05-25 | 2019-10-28 | Univ California | Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| ITTO20130669A1 (it) * | 2013-08-05 | 2015-02-06 | Consiglio Nazionale Ricerche | Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari |
| WO2016153012A1 (ja) | 2015-03-24 | 2016-09-29 | 協和発酵キリン株式会社 | 核酸含有脂質ナノ粒子 |
| EP3332008A4 (en) * | 2015-09-23 | 2019-01-16 | Université Laval | CHANGE OF THE DYSTROPHINE GENE AND USES THEREOF |
| JP6998313B2 (ja) * | 2016-02-11 | 2022-02-04 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 細胞のゲノムにおける変異ジストロフィン遺伝子を修飾する方法及び組成物 |
| US11674128B2 (en) * | 2016-12-12 | 2023-06-13 | Tsinghua University | Engineering of a minimal SaCas9 CRISPR/Cas system for gene editing and transcriptional regulation optimized by enhanced guide RNA |
| EP3579858A4 (en) * | 2017-02-07 | 2020-12-23 | The Regents of The University of California | GENE THERAPY AGAINST HAPLOINSUFFICIENCY |
| US11674138B2 (en) * | 2017-03-13 | 2023-06-13 | President And Fellows Of Harvard College | Methods of modulating expression of target nucleic acid sequences in a cell |
| US20210254056A1 (en) * | 2017-05-05 | 2021-08-19 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
| CA3097375A1 (en) | 2018-04-27 | 2019-10-31 | Universitat Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| WO2019236081A1 (en) * | 2018-06-06 | 2019-12-12 | Salk Institute For Biological Studies | Targeted gene activation using modified guide rna |
-
2019
- 2019-11-15 MX MX2021005720A patent/MX2021005720A/es unknown
- 2019-11-15 WO PCT/JP2019/045716 patent/WO2020101042A1/en not_active Ceased
- 2019-11-15 EP EP19824401.4A patent/EP3880255A1/en active Pending
- 2019-11-15 AU AU2019381460A patent/AU2019381460A1/en not_active Abandoned
- 2019-11-15 BR BR112021009481-4A patent/BR112021009481A2/pt not_active IP Right Cessation
- 2019-11-15 TW TW108141671A patent/TW202033224A/zh unknown
- 2019-11-15 CN CN201980075173.0A patent/CN113271982B/zh active Active
- 2019-11-15 KR KR1020217018333A patent/KR102931423B1/ko active Active
- 2019-11-15 IL IL283123A patent/IL283123B2/en unknown
- 2019-11-15 CA CA3119618A patent/CA3119618A1/en active Pending
- 2019-11-15 SG SG11202105030VA patent/SG11202105030VA/en unknown
- 2019-11-15 JP JP2021551108A patent/JP7069426B2/ja active Active
-
2021
- 2021-05-11 PH PH12021551122A patent/PH12021551122A1/en unknown
- 2021-05-14 US US17/320,643 patent/US11473071B2/en active Active
- 2021-05-26 CO CONC2021/0006957A patent/CO2021006957A2/es unknown
-
2022
- 2022-05-02 JP JP2022076261A patent/JP2022115976A/ja active Pending