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 (https=) | 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 (https=) |
| EP (1) | EP3565897B1 (https=) |
| JP (1) | JP2020503869A (https=) |
| KR (1) | KR102606174B1 (https=) |
| CN (1) | CN110506115A (https=) |
| AU (1) | AU2018205521A1 (https=) |
| BR (1) | BR112019013962A2 (https=) |
| CA (1) | CA3048635A1 (https=) |
| CL (1) | CL2019001882A1 (https=) |
| CO (1) | CO2019008181A2 (https=) |
| CR (1) | CR20190326A (https=) |
| DK (1) | DK3565897T3 (https=) |
| DO (1) | DOP2019000179A (https=) |
| EC (1) | ECSP19056408A (https=) |
| IL (1) | IL267786A (https=) |
| JO (1) | JOP20190166A1 (https=) |
| MA (1) | MA47239A (https=) |
| MX (1) | MX2019008064A (https=) |
| PE (1) | PE20191357A1 (https=) |
| PH (1) | PH12019501561A1 (https=) |
| SG (1) | SG11201906147VA (https=) |
| WO (1) | WO2018129296A1 (https=) |
Families Citing this family (37)
| 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 |
| 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 |
| EP3617311B1 (en) * | 2017-03-30 | 2024-11-20 | Kyoto University | Method for inducing exon skipping by genome editing |
| 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 |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| EP3810775A1 (en) * | 2018-06-21 | 2021-04-28 | 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| BR112021017655A2 (pt) * | 2019-03-07 | 2021-11-16 | Univ Columbia | Método e sistema de integração de dna guiada por rna usando transposons tipo tn7 |
| US20220177876A1 (en) * | 2019-04-12 | 2022-06-09 | The Regents Of The University Of California | Compositions and Methods for Modifying Dystrophin Genes |
| JP2022526669A (ja) * | 2019-04-12 | 2022-05-25 | 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 |
| US20210047649A1 (en) | 2019-05-08 | 2021-02-18 | 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 |
| 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 |
| WO2021072276A1 (en) * | 2019-10-11 | 2021-04-15 | 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 |
| EP4103214B1 (en) * | 2020-02-13 | 2026-04-29 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| 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 |
| 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 |
| AU2021342057A1 (en) * | 2020-09-09 | 2023-04-06 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of duchenne muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| JP2024525608A (ja) | 2021-07-09 | 2024-07-12 | ダイン セラピューティクス，インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体および製剤 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2023159103A1 (en) * | 2022-02-17 | 2023-08-24 | The Board Of Regents Of The University Of Texas System | CRISPR/SpCas9 VARIANT AND METHODS FOR ENHANCED CORRECTON OF DUCHENNE MUSCULAR DYSTROPHY MUTATIONS |
| KR20230134098A (ko) * | 2022-03-10 | 2023-09-20 | 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 |
|---|---|---|---|---|
| DK1737966T3 (da) * | 2004-04-02 | 2012-07-09 | Univ Texas | Cancerspecifik promotorer |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| EP4659765A3 (en) * | 2014-03-12 | 2026-02-18 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| CN106714845A (zh) * | 2014-08-11 | 2017-05-24 | 得克萨斯州大学系统董事会 | 通过crispr/cas9介导的基因编辑预防肌营养不良 |
| US20170266320A1 (en) * | 2014-12-01 | 2017-09-21 | President And Fellows Of Harvard College | RNA-Guided Systems for In Vivo Gene Editing |
| JP6832280B2 (ja) * | 2015-01-16 | 2021-02-24 | ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| KR102888521B1 (ko) * | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| WO2016174056A1 (en) * | 2015-04-27 | 2016-11-03 | Genethon | Compositions and methods for the treatment of nucleotide repeat expansion disorders |
| 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 |
| 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 WO PCT/US2018/012558 patent/WO2018129296A1/en not_active Ceased
- 2018-01-05 SG SG11201906147VA patent/SG11201906147VA/en unknown
- 2018-01-05 MX MX2019008064A patent/MX2019008064A/es unknown
- 2018-01-05 MA MA047239A patent/MA47239A/fr unknown
- 2018-01-05 EP EP18704637.0A patent/EP3565897B1/en active Active
- 2018-01-05 CN CN201880010288.7A patent/CN110506115A/zh active Pending
- 2018-01-05 CR CR20190326A patent/CR20190326A/es unknown
- 2018-01-05 BR BR112019013962-1A patent/BR112019013962A2/pt not_active IP Right Cessation
- 2018-01-05 PE PE2019001384A patent/PE20191357A1/es unknown
- 2018-01-05 CA CA3048635A patent/CA3048635A1/en active Pending
- 2018-01-05 KR KR1020197022753A patent/KR102606174B1/ko active Active
- 2018-01-05 DK DK18704637.0T patent/DK3565897T3/da active
- 2018-01-05 JP JP2019536508A patent/JP2020503869A/ja active Pending
- 2018-01-05 US US16/476,137 patent/US20190338311A1/en not_active Abandoned
- 2018-01-05 AU AU2018205521A patent/AU2018205521A1/en not_active Abandoned
-
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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