JP2020503869A5 - - Google Patents
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- JP2020503869A5 JP2020503869A5 JP2019536508A JP2019536508A JP2020503869A5 JP 2020503869 A5 JP2020503869 A5 JP 2020503869A5 JP 2019536508 A JP2019536508 A JP 2019536508A JP 2019536508 A JP2019536508 A JP 2019536508A JP 2020503869 A5 JP2020503869 A5 JP 2020503869A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- sequence encoding
- nucleic acid
- guide rna
- sequence
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims description 118
- 108020004707 nucleic acids Proteins 0.000 claims description 110
- 102000039446 nucleic acids Human genes 0.000 claims description 110
- 102100024108 Dystrophin Human genes 0.000 claims description 65
- 108020005004 Guide RNA Proteins 0.000 claims description 64
- 239000013598 vector Substances 0.000 claims description 58
- 239000000203 mixture Substances 0.000 claims description 44
- 108010069091 Dystrophin Proteins 0.000 claims description 27
- 108091033409 CRISPR Proteins 0.000 claims description 21
- 101710163270 Nuclease Proteins 0.000 claims description 21
- 201000006938 muscular dystrophy Diseases 0.000 claims description 15
- 210000003205 muscle Anatomy 0.000 claims description 14
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 13
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 11
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 10
- 241000702421 Dependoparvovirus Species 0.000 claims description 10
- 239000013603 viral vector Substances 0.000 claims description 9
- 230000001939 inductive effect Effects 0.000 claims description 5
- 241000191967 Staphylococcus aureus Species 0.000 claims description 2
- 241000193996 Streptococcus pyogenes Species 0.000 claims description 2
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- -1 DMD guide RNA (gRNA) Chemical class 0.000 claims 1
- 238000000034 method Methods 0.000 description 45
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 description 33
- 210000004027 cell Anatomy 0.000 description 27
- 239000013607 AAV vector Substances 0.000 description 26
- 102000001039 Dystrophin Human genes 0.000 description 22
- 208000024891 symptom Diseases 0.000 description 10
- 210000001087 myotubule Anatomy 0.000 description 6
- 239000002773 nucleotide Substances 0.000 description 6
- 125000003729 nucleotide group Chemical group 0.000 description 6
- 210000005260 human cell Anatomy 0.000 description 5
- 230000002950 deficient Effects 0.000 description 4
- 230000010076 replication Effects 0.000 description 4
- 210000001519 tissue Anatomy 0.000 description 4
- 239000003937 drug carrier Substances 0.000 description 3
- 210000004962 mammalian cell Anatomy 0.000 description 3
- 230000000926 neurological effect Effects 0.000 description 3
- 230000000750 progressive effect Effects 0.000 description 3
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 2
- 241000649045 Adeno-associated virus 10 Species 0.000 description 2
- 241000649046 Adeno-associated virus 11 Species 0.000 description 2
- 108700024394 Exon Proteins 0.000 description 2
- 208000010428 Muscle Weakness Diseases 0.000 description 2
- 206010028372 Muscular weakness Diseases 0.000 description 2
- 206010033799 Paralysis Diseases 0.000 description 2
- 230000007812 deficiency Effects 0.000 description 2
- 101150015424 dmd gene Proteins 0.000 description 2
- 239000007924 injection Substances 0.000 description 2
- 238000002347 injection Methods 0.000 description 2
- 238000003780 insertion Methods 0.000 description 2
- 230000037431 insertion Effects 0.000 description 2
- 210000000663 muscle cell Anatomy 0.000 description 2
- 239000013612 plasmid Substances 0.000 description 2
- 210000002966 serum Anatomy 0.000 description 2
- 210000001057 smooth muscle myoblast Anatomy 0.000 description 2
- 241000282412 Homo Species 0.000 description 1
- 101100443349 Homo sapiens DMD gene Proteins 0.000 description 1
- 201000006347 Intellectual Disability Diseases 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 208000034819 Mobility Limitation Diseases 0.000 description 1
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 1
- 208000000875 Spinal Curvatures Diseases 0.000 description 1
- 230000002159 abnormal effect Effects 0.000 description 1
- 150000003838 adenosines Chemical class 0.000 description 1
- 125000003275 alpha amino acid group Chemical group 0.000 description 1
- 230000014461 bone development Effects 0.000 description 1
- 210000004899 c-terminal region Anatomy 0.000 description 1
- 230000009194 climbing Effects 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 230000001747 exhibiting effect Effects 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 210000005003 heart tissue Anatomy 0.000 description 1
- 238000001802 infusion Methods 0.000 description 1
- 239000007925 intracardiac injection Substances 0.000 description 1
- 239000007927 intramuscular injection Substances 0.000 description 1
- 238000010255 intramuscular injection Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 230000007775 late Effects 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 210000004197 pelvis Anatomy 0.000 description 1
- 210000003314 quadriceps muscle Anatomy 0.000 description 1
- 210000002027 skeletal muscle Anatomy 0.000 description 1
- 230000005945 translocation Effects 0.000 description 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762442606P | 2017-01-05 | 2017-01-05 | |
| US62/442,606 | 2017-01-05 | ||
| US201762544449P | 2017-08-11 | 2017-08-11 | |
| US62/544,449 | 2017-08-11 | ||
| US201762596298P | 2017-12-08 | 2017-12-08 | |
| US62/596,298 | 2017-12-08 | ||
| PCT/US2018/012558 WO2018129296A1 (en) | 2017-01-05 | 2018-01-05 | Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020503869A JP2020503869A (ja) | 2020-02-06 |
| JP2020503869A5 true JP2020503869A5 (enExample) | 2021-02-12 |
Family
ID=61193018
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019536508A Pending JP2020503869A (ja) | 2017-01-05 | 2018-01-05 | トリプルガイド配列と共にｃｒｉｓｐｒ／ｃａｓ９を用いるエクソンスキッピング改変のための最適化戦略 |
Country Status (22)
| Country | Link |
|---|---|
| US (1) | US20190338311A1 (enExample) |
| EP (1) | EP3565897B1 (enExample) |
| JP (1) | JP2020503869A (enExample) |
| KR (1) | KR102606174B1 (enExample) |
| CN (1) | CN110506115A (enExample) |
| AU (1) | AU2018205521A1 (enExample) |
| BR (1) | BR112019013962A2 (enExample) |
| CA (1) | CA3048635A1 (enExample) |
| CL (1) | CL2019001882A1 (enExample) |
| CO (1) | CO2019008181A2 (enExample) |
| CR (1) | CR20190326A (enExample) |
| DK (1) | DK3565897T3 (enExample) |
| DO (1) | DOP2019000179A (enExample) |
| EC (1) | ECSP19056408A (enExample) |
| IL (1) | IL267786A (enExample) |
| JO (1) | JOP20190166A1 (enExample) |
| MA (1) | MA47239A (enExample) |
| MX (1) | MX2019008064A (enExample) |
| PE (1) | PE20191357A1 (enExample) |
| PH (1) | PH12019501561A1 (enExample) |
| SG (1) | SG11201906147VA (enExample) |
| WO (1) | WO2018129296A1 (enExample) |
Families Citing this family (39)
| 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 |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| 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 |
| CN110892069B (zh) * | 2017-03-30 | 2024-02-09 | 国立大学法人京都大学 | 基于基因组编辑的外显子跳跃诱导方法 |
| EP3735462A1 (en) * | 2018-01-05 | 2020-11-11 | The Board of Regents of The University of Texas System | Therapeutic crispr/cas9 compositions and methods of use |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| WO2019246480A1 (en) * | 2018-06-21 | 2019-12-26 | The Board Of Regents Of The University Of Texas System | Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| EP3830259A4 (en) | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY |
| WO2020122104A1 (ja) * | 2018-12-11 | 2020-06-18 | 国立大学法人京都大学 | ゲノムｄｎａに欠失を誘導する方法 |
| WO2020142714A1 (en) * | 2019-01-04 | 2020-07-09 | Exonics Therapeutics, Inc. | Aav expression cassette and aav vectors comprising the same |
| WO2020181264A1 (en) | 2019-03-07 | 2020-09-10 | The Trustees Of Columbia University In The City Of New York | Rna-guided dna integration using tn7-like transposons |
| WO2020210214A1 (en) * | 2019-04-12 | 2020-10-15 | The Regents Of The University Of California | Compositions and methods for modifying dystrophin genes |
| JP7881154B2 (ja) * | 2019-04-12 | 2026-06-29 | デューク ユニバーシティ | ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物 |
| 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 |
| EP3966327A1 (en) | 2019-05-08 | 2022-03-16 | Vertex Pharmaceuticals Incorporated | Crispr/cas all-in-two vector systems for treatment of dmd |
| KR102264115B1 (ko) * | 2019-05-10 | 2021-06-14 | 한국과학기술연구원 | 무-운반체 다중 CRISPR/Cas 9 유전자 편집 복합체 및 그의 용도 |
| RU2722934C1 (ru) | 2019-06-11 | 2020-06-05 | Автономная некоммерческая образовательная организация высшего образования Сколковский институт науки и технологий | Средство разрезания днк на основе cas9 белка из бактерии pasteurella pneumotropica |
| CN110499333A (zh) * | 2019-08-01 | 2019-11-26 | 广州德赫生物科技有限公司 | 用于修复dmd基因突变的核酸序列及系统 |
| US20240091379A1 (en) * | 2019-10-11 | 2024-03-21 | Yale University | Compositions and methods for upregulating isoforms of dystrophin as therapy for duchenne muscular dystrophy (dmd) |
| CN114846146B (zh) * | 2019-10-29 | 2024-04-12 | 基恩科雷有限责任公司 | 用于增加CRISPR/Cas12f1系统的效率的工程化引导RNA及其用途 |
| FI4103214T3 (fi) * | 2020-02-13 | 2026-07-03 | Tenaya Therapeutics Inc | Geeniterapian vektorit sydänsairauden hoitoon |
| CN111172191B (zh) * | 2020-02-21 | 2020-12-22 | 浙江大学 | 一种高效基因敲除载体及其应用 |
| EP4125349A4 (en) * | 2020-04-27 | 2024-07-10 | Duke University | GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| CN115011598A (zh) * | 2020-09-02 | 2022-09-06 | 西湖大学 | 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用 |
| CA3193753A1 (en) * | 2020-09-09 | 2022-03-17 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of duchenne muscular dystrophy |
| WO2023283623A1 (en) | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| EP4479536A1 (en) * | 2022-02-17 | 2024-12-25 | The Board Of Regents Of The University Of Texas System | Crispr/spcas9 variant and methods for enhanced correcton of duchenne muscular dystrophy mutations |
| WO2023172116A1 (ko) * | 2022-03-10 | 2023-09-14 | 주식회사 진코어 | 듀센 근이영양증 치료를 위한 유전자 편집 시스템 및 이를 이용한 질병 치료 방법 |
| 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 (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1737966B1 (en) * | 2004-04-02 | 2012-05-09 | Board of Regents, The University of Texas System | Cancer specific promoters |
| 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 |
| AU2015229299A1 (en) * | 2014-03-12 | 2016-09-22 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| US20160058889A1 (en) * | 2014-08-11 | 2016-03-03 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cas9-mediated gene editing |
| WO2016089866A1 (en) * | 2014-12-01 | 2016-06-09 | President And Fellows Of Harvard College | Rna-guided systems for in vivo gene editing |
| WO2016115543A2 (en) * | 2015-01-16 | 2016-07-21 | University Of Washington | Novel micro-dystrophins and related methods of use |
| CN107787367B (zh) * | 2015-04-06 | 2021-10-26 | 里兰斯坦福初级大学理事会 | 用于crispr/cas介导的基因调控的化学修饰的引导rna |
| US11104897B2 (en) * | 2015-04-27 | 2021-08-31 | Genethon | Compositions and methods for the treatment of nucleotide repeat expansion disorders |
| EP4279084B1 (en) * | 2015-10-28 | 2025-06-11 | Vertex Pharmaceuticals Inc. | Materials and methods for treatment of duchenne muscular dystrophy |
| 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 |
| US20170362635A1 (en) * | 2016-06-20 | 2017-12-21 | University Of Washington | Muscle-specific crispr/cas9 editing of genes |
-
2017
- 2017-06-16 JO JOP/2019/0166A patent/JOP20190166A1/ar unknown
-
2018
- 2018-01-05 KR KR1020197022753A patent/KR102606174B1/ko active Active
- 2018-01-05 CR CR20190326A patent/CR20190326A/es unknown
- 2018-01-05 PE PE2019001384A patent/PE20191357A1/es unknown
- 2018-01-05 SG SG11201906147VA patent/SG11201906147VA/en unknown
- 2018-01-05 EP EP18704637.0A patent/EP3565897B1/en active Active
- 2018-01-05 MA MA047239A patent/MA47239A/fr unknown
- 2018-01-05 MX MX2019008064A patent/MX2019008064A/es unknown
- 2018-01-05 JP JP2019536508A patent/JP2020503869A/ja active Pending
- 2018-01-05 CA CA3048635A patent/CA3048635A1/en active Pending
- 2018-01-05 CN CN201880010288.7A patent/CN110506115A/zh active Pending
- 2018-01-05 DK DK18704637.0T patent/DK3565897T3/da active
- 2018-01-05 AU AU2018205521A patent/AU2018205521A1/en not_active Abandoned
- 2018-01-05 US US16/476,137 patent/US20190338311A1/en not_active Abandoned
- 2018-01-05 WO PCT/US2018/012558 patent/WO2018129296A1/en not_active Ceased
- 2018-01-05 BR BR112019013962-1A patent/BR112019013962A2/pt not_active IP Right Cessation
-
2019
- 2019-07-02 IL IL267786A patent/IL267786A/en unknown
- 2019-07-02 PH PH12019501561A patent/PH12019501561A1/en unknown
- 2019-07-05 CL CL2019001882A patent/CL2019001882A1/es unknown
- 2019-07-05 DO DO2019000179A patent/DOP2019000179A/es unknown
- 2019-07-29 CO CO2019008181A patent/CO2019008181A2/es unknown
- 2019-08-06 EC ECSENADI201956408A patent/ECSP19056408A/es unknown
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