JP2022115976A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- 108091033409 CRISPR Proteins 0.000 claims 1
- 238000010354 CRISPR gene editing Methods 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 101710163270 Nuclease Proteins 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 239000012636 effector Substances 0.000 claims 1
- 108020001507 fusion proteins Proteins 0.000 claims 1
- 102000037865 fusion proteins Human genes 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- 108091006106 transcriptional activators Proteins 0.000 claims 1
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 (enExample) | 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 (enExample) |
| EP (1) | EP3880255A1 (enExample) |
| JP (2) | JP7069426B2 (enExample) |
| KR (1) | KR20210093954A (enExample) |
| CN (1) | CN113271982B (enExample) |
| AU (1) | AU2019381460A1 (enExample) |
| BR (1) | BR112021009481A2 (enExample) |
| CA (1) | CA3119618A1 (enExample) |
| CO (1) | CO2021006957A2 (enExample) |
| IL (1) | IL283123B1 (enExample) |
| MX (1) | MX2021005720A (enExample) |
| PH (1) | PH12021551122A1 (enExample) |
| SG (1) | SG11202105030VA (enExample) |
| TW (1) | TW202033224A (enExample) |
| WO (1) | WO2020101042A1 (enExample) |
Families Citing this family (12)
| 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 |
| CN117534769A (zh) | 2018-04-19 | 2024-02-09 | 加利福尼亚大学董事会 | 用于基因编辑的组合物和方法 |
| CA3103088A1 (en) * | 2018-06-08 | 2019-12-12 | Modalis Therapeutics Corporation | Modified cas9 protein and use thereof |
| EP3833758A4 (en) * | 2018-08-07 | 2022-05-18 | Modalis Therapeutics Corporation | NOVEL TRANSCRIPTION ACTIVATOR |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| CN116323941A (zh) * | 2020-09-29 | 2023-06-23 | 吉尼松公司 | 通过诱导抗肌萎缩蛋白相关蛋白调节元件内的突变增强细胞中抗肌萎缩蛋白相关蛋白表达及其治疗用途 |
| 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 |
| 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 |
|---|---|---|---|---|
| CA2407309C (en) * | 2000-04-28 | 2011-08-02 | Xiao Xiao | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| EP2017338A1 (en) | 2001-05-24 | 2009-01-21 | Genzyme Corporation | Muscle-specific expression vectors |
| SG168422A1 (en) | 2001-11-13 | 2011-02-28 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP3910063A1 (en) | 2003-09-30 | 2021-11-17 | 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 |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| KR20160074368A (ko) * | 2012-05-16 | 2016-06-28 | 라나 테라퓨틱스, 인크. | Utrn 발현을 조절하기 위한 조성물 및 방법 |
| SI3401400T1 (sl) | 2012-05-25 | 2019-10-30 | Univ California | Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja |
| KR20230136697A (ko) * | 2013-06-05 | 2023-09-26 | 듀크 유니버시티 | Rna-가이드 유전자 편집 및 유전자 조절 |
| 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 |
| KR20170130531A (ko) | 2015-03-24 | 2017-11-28 | 교와 핫꼬 기린 가부시키가이샤 | 핵산 함유 지질 나노 입자 |
| CA2996982A1 (en) * | 2015-09-23 | 2017-03-30 | Universite Laval | Modification of the dystrophin gene and uses thereof |
| US11666666B2 (en) * | 2016-02-11 | 2023-06-06 | 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 |
| EP3579858A4 (en) * | 2017-02-07 | 2020-12-23 | The Regents of The University of California | GENE THERAPY AGAINST HAPLOINSUFFICIENCY |
| WO2018169983A1 (en) * | 2017-03-13 | 2018-09-20 | 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 |
| 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 CN CN201980075173.0A patent/CN113271982B/zh active Active
- 2019-11-15 JP JP2021551108A patent/JP7069426B2/ja active Active
- 2019-11-15 BR BR112021009481-4A patent/BR112021009481A2/pt not_active IP Right Cessation
- 2019-11-15 CA CA3119618A patent/CA3119618A1/en active Pending
- 2019-11-15 WO PCT/JP2019/045716 patent/WO2020101042A1/en not_active Ceased
- 2019-11-15 MX MX2021005720A patent/MX2021005720A/es unknown
- 2019-11-15 TW TW108141671A patent/TW202033224A/zh unknown
- 2019-11-15 AU AU2019381460A patent/AU2019381460A1/en not_active Abandoned
- 2019-11-15 EP EP19824401.4A patent/EP3880255A1/en active Pending
- 2019-11-15 KR KR1020217018333A patent/KR20210093954A/ko active Pending
- 2019-11-15 SG SG11202105030VA patent/SG11202105030VA/en unknown
-
2021
- 2021-05-11 PH PH12021551122A patent/PH12021551122A1/en unknown
- 2021-05-12 IL IL283123A patent/IL283123B1/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