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
Links
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 (https=) | 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)
| Country | Link |
|---|---|
| US (1) | US11473071B2 (https=) |
| EP (1) | EP3880255A1 (https=) |
| JP (2) | JP7069426B2 (https=) |
| KR (1) | KR102931423B1 (https=) |
| CN (1) | CN113271982B (https=) |
| AU (1) | AU2019381460A1 (https=) |
| BR (1) | BR112021009481A2 (https=) |
| CA (1) | CA3119618A1 (https=) |
| CO (1) | CO2021006957A2 (https=) |
| IL (1) | IL283123B2 (https=) |
| MX (1) | MX2021005720A (https=) |
| PH (1) | PH12021551122A1 (https=) |
| SG (1) | SG11202105030VA (https=) |
| TW (1) | TW202033224A (https=) |
| WO (1) | WO2020101042A1 (https=) |
Families Citing this family (13)
| 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. |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| WO2019144061A1 (en) | 2018-01-19 | 2019-07-25 | Duke University | Genome engineering with crispr-cas systems in eukaryotes |
| JP7555822B2 (ja) | 2018-04-19 | 2024-09-25 | ザ・リージエンツ・オブ・ザ・ユニバーシテイー・オブ・カリフオルニア | ゲノム編集のための組成物および方法 |
| CA3103088A1 (en) * | 2018-06-08 | 2019-12-12 | Modalis Therapeutics Corporation | Modified cas9 protein and use thereof |
| CN112585266A (zh) * | 2018-08-07 | 2021-03-30 | 摩大力斯医疗株式会社 | 新型转录激活物 |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| WO2022069598A1 (en) | 2020-09-29 | 2022-04-07 | Genethon | Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements and therapeutic use thereof |
| KR102421411B1 (ko) | 2021-10-29 | 2022-07-18 | 주식회사 케이에스비튜젠 | 옥시라세탐을 포함하는 근이영양증 예방 또는 치료용 조성물 |
| EP4331579A4 (en) * | 2021-04-26 | 2025-04-09 | KSB Tugen Inc. | Composition comprising oxiracetam for preventing or treating muscular disease |
| 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 |
| 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 |
|---|---|---|---|---|
| ATE437944T1 (de) * | 2000-04-28 | 2009-08-15 | Asklepios Biopharmaceutical In | Für das dystrophin-minigen kodierende dna- sequenzen und verfahren zu deren verwendung |
| EP2017338A1 (en) | 2001-05-24 | 2009-01-21 | Genzyme Corporation | Muscle-specific expression vectors |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| CA2553676A1 (en) | 2004-01-28 | 2005-08-11 | Research Development Foundation | 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 |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| BR112014028634A2 (pt) * | 2012-05-16 | 2017-06-27 | Rana Therapeutics Inc | composições e métodos para modulação da expressão de utrn |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| EP3004370B1 (en) * | 2013-06-05 | 2024-08-21 | 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 |
| AU2016237148A1 (en) | 2015-03-24 | 2017-11-23 | Kyowa Hakko Kirin Co., Ltd. | Nucleic acid-containing lipid nanoparticles |
| US20180265859A1 (en) * | 2015-09-23 | 2018-09-20 | UNIVERSITé LAVAL | Modification of the dystrophin gene and uses thereof |
| 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 |
| 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 |
| JP2020506948A (ja) * | 2017-02-07 | 2020-03-05 | 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 | ハプロ不全のための遺伝子治療 |
| 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 |
| WO2018204764A1 (en) * | 2017-05-05 | 2018-11-08 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
| AU2019258830B2 (en) | 2018-04-27 | 2025-12-18 | Universität 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 EP EP19824401.4A patent/EP3880255A1/en active Pending
- 2019-11-15 MX MX2021005720A patent/MX2021005720A/es unknown
- 2019-11-15 AU AU2019381460A patent/AU2019381460A1/en not_active Abandoned
- 2019-11-15 IL IL283123A patent/IL283123B2/en unknown
- 2019-11-15 WO PCT/JP2019/045716 patent/WO2020101042A1/en not_active Ceased
- 2019-11-15 BR BR112021009481-4A patent/BR112021009481A2/pt not_active IP Right Cessation
- 2019-11-15 CN CN201980075173.0A patent/CN113271982B/zh active Active
- 2019-11-15 TW TW108141671A patent/TW202033224A/zh unknown
- 2019-11-15 JP JP2021551108A patent/JP7069426B2/ja active Active
- 2019-11-15 SG SG11202105030VA patent/SG11202105030VA/en unknown
- 2019-11-15 CA CA3119618A patent/CA3119618A1/en active Pending
- 2019-11-15 KR KR1020217018333A patent/KR102931423B1/ko 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