JP2019515914A5 - - Google Patents
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- Publication number
- JP2019515914A5 JP2019515914A5 JP2018555262A JP2018555262A JP2019515914A5 JP 2019515914 A5 JP2019515914 A5 JP 2019515914A5 JP 2018555262 A JP2018555262 A JP 2018555262A JP 2018555262 A JP2018555262 A JP 2018555262A JP 2019515914 A5 JP2019515914 A5 JP 2019515914A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nuclease
- guide rna
- guide
- guides
- 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
- 229920002391 Guide RNA Polymers 0.000 claims description 195
- 108020005004 Guide RNA Proteins 0.000 claims description 195
- 101700080605 NUC1 Proteins 0.000 claims description 182
- 101700006494 nucA Proteins 0.000 claims description 182
- 239000000203 mixture Substances 0.000 claims description 99
- RWQNBRDOKXIBIV-UHFFFAOYSA-N Thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 16
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 16
- 239000002773 nucleotide Substances 0.000 claims description 16
- 125000003729 nucleotide group Chemical group 0.000 claims description 16
- 230000000295 complement Effects 0.000 claims description 13
- 230000035772 mutation Effects 0.000 claims description 13
- 239000000825 pharmaceutical preparation Substances 0.000 claims description 12
- 102220005941 rs80358192 Human genes 0.000 claims description 12
- 102200030577 COL8A2 Q455K Human genes 0.000 claims description 9
- 102000009523 Transcription Factor 4 Human genes 0.000 claims description 9
- 108010048992 Transcription Factor 4 Proteins 0.000 claims description 9
- 102220055955 rs727504229 Human genes 0.000 claims description 9
- 102100001026 COL8A2 Human genes 0.000 claims description 8
- 101710039321 COL8A2 Proteins 0.000 claims description 8
- 229940113082 Thymine Drugs 0.000 claims description 8
- 229940035893 Uracil Drugs 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 7
- 108090000623 proteins and genes Proteins 0.000 claims description 7
- 238000011105 stabilization Methods 0.000 claims description 7
- 206010008609 Cholangitis sclerosing Diseases 0.000 claims description 6
- 201000000742 primary sclerosing cholangitis Diseases 0.000 claims description 6
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims description 4
- 241000282485 Vulpes vulpes Species 0.000 claims description 4
- 201000004180 corneal endothelial dystrophy Diseases 0.000 claims description 4
- 206010011005 Corneal dystrophy Diseases 0.000 claims description 3
- 229940012356 Eye Drops Drugs 0.000 claims description 3
- 239000003814 drug Substances 0.000 claims description 3
- 239000003889 eye drop Substances 0.000 claims description 3
- 150000002632 lipids Chemical class 0.000 claims description 3
- 238000000386 microscopy Methods 0.000 claims description 3
- 239000002105 nanoparticle Substances 0.000 claims description 3
- 239000002674 ointment Substances 0.000 claims description 3
- 238000002360 preparation method Methods 0.000 claims description 3
- 201000000980 schizophrenia Diseases 0.000 claims description 3
- 230000003612 virological Effects 0.000 claims description 3
- 229920000033 CRISPR Polymers 0.000 claims description 2
- 108010082319 CRISPR-Associated Protein 9 Proteins 0.000 claims description 2
- 238000010453 CRISPR/Cas method Methods 0.000 claims description 2
- 102000001191 Collagen Type VIII Human genes 0.000 claims description 2
- 108010069526 Collagen Type VIII Proteins 0.000 claims description 2
- 229920001850 Nucleic acid sequence Polymers 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 238000009472 formulation Methods 0.000 claims 8
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 230000004452 decreased vision Effects 0.000 claims 1
- 102000003995 transcription factors Human genes 0.000 claims 1
- 108090000464 transcription factors Proteins 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662326700P | 2016-04-22 | 2016-04-22 | |
| US62/326,700 | 2016-04-22 | ||
| PCT/US2017/028981 WO2017185054A1 (en) | 2016-04-22 | 2017-04-21 | Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019515914A JP2019515914A (ja) | 2019-06-13 |
| JP2019515914A5 true JP2019515914A5 (US07794700-20100914-C00152.png) | 2020-06-11 |
Family
ID=58692575
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018555262A Pending JP2019515914A (ja) | 2016-04-22 | 2017-04-21 | 転写因子４内のトリヌクレオチドリピートと関連する疾患の治療のための組成物および方法 |
Country Status (11)
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN108350446A (zh) * | 2015-07-02 | 2018-07-31 | 约翰霍普金斯大学 | 基于crispr/cas9的治疗 |
| WO2022072458A1 (en) * | 2020-09-29 | 2022-04-07 | Avellino Lab Usa, Inc. | Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy |
| WO2018165541A1 (en) * | 2017-03-10 | 2018-09-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
| US20230201373A1 (en) * | 2017-12-15 | 2023-06-29 | Regents Of The University Of Minnesota | Crispr-mediated genome editing with vectors |
| CN109929872A (zh) * | 2017-12-18 | 2019-06-25 | 中国科学院遗传与发育生物学研究所 | 一种通过基因编辑技术创制番茄白果材料的方法 |
| US11891635B2 (en) | 2017-12-21 | 2024-02-06 | Max-Delbrück-Centrum Für Molekulare Medizin In Der Helmholtz-Gemeinschaft | Nucleic acid sequence replacement by NHEJ |
| US20190151470A1 (en) | 2018-01-21 | 2019-05-23 | RoverMed BioSciences, LLC | Nanoparticles comprising protein-polynucleotide complexes and for delivering protein based complexes |
| CA3131919A1 (en) | 2019-02-28 | 2020-09-03 | Regeneron Pharmaceuticals, Inc. | Adeno-associated virus vectors for the delivery of therapeutics |
| CN111944813B (zh) * | 2020-08-24 | 2022-07-15 | 武汉纽福斯生物科技有限公司 | 核苷酸、病毒载体及其应用和RNAi药物制剂 |
| CN111944814B (zh) * | 2020-08-24 | 2022-07-15 | 武汉纽福斯生物科技有限公司 | 寡核苷酸、病毒载体及其应用和RNAi药物制剂 |
| IL303506A (en) | 2020-12-11 | 2023-08-01 | Intellia Therapeutics Inc | Polynucleotides, compounds and methods for genome editing involving deamination |
| WO2023086842A1 (en) * | 2021-11-09 | 2023-05-19 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006007712A1 (en) | 2004-07-19 | 2006-01-26 | Protiva Biotherapeutics, Inc. | Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents |
| WO2013101711A1 (en) * | 2011-12-30 | 2013-07-04 | Mayo Foundation For Medical Education And Research | Assessing likelihood of developing fuchs' corneal dystrophy |
| MX362866B (es) * | 2012-05-25 | 2019-02-20 | Univ California | Metodos y composiciones para la modificacion de adn objetivo dirigida por arn y para la modulacion de la transcripcion dirigida por arn. |
| WO2014093701A1 (en) * | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| ES2887254T3 (es) | 2013-03-08 | 2021-12-22 | Novartis Ag | Lípidos y composiciones lipídicas para el suministro de agentes activos |
| KR102380245B1 (ko) * | 2013-11-07 | 2022-03-30 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| BR112016013213A2 (pt) * | 2013-12-12 | 2017-12-05 | Massachusetts Inst Technology | administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para visar distúrbios e doenças usando componentes de administração de partículas |
| ES2774968T3 (es) | 2013-12-19 | 2020-07-23 | Novartis Ag | Lípidos y composiciones lipídicas para la administración de agentes activos |
| AU2015247323B2 (en) * | 2014-04-18 | 2021-07-01 | Editas Medicine, Inc. | CRISPR-Cas-related methods, compositions and components for cancer immunotherapy |
| CN108350446A (zh) * | 2015-07-02 | 2018-07-31 | 约翰霍普金斯大学 | 基于crispr/cas9的治疗 |
-
2017
- 2017-04-21 JP JP2018555262A patent/JP2019515914A/ja active Pending
- 2017-04-21 US US16/095,236 patent/US20190142972A1/en not_active Abandoned
- 2017-04-21 CN CN201780036135.5A patent/CN109414450A/zh active Pending
- 2017-04-21 CA CA3021647A patent/CA3021647A1/en active Pending
- 2017-04-21 EP EP17722552.1A patent/EP3445375A1/en not_active Withdrawn
- 2017-04-21 WO PCT/US2017/028981 patent/WO2017185054A1/en active Application Filing
- 2017-04-21 AU AU2017254718A patent/AU2017254718A1/en not_active Abandoned
- 2017-04-21 BR BR112018071439-9A patent/BR112018071439A2/pt not_active IP Right Cessation
- 2017-04-21 KR KR1020187033525A patent/KR20180134412A/ko not_active Application Discontinuation
- 2017-04-21 MX MX2018012873A patent/MX2018012873A/es unknown
-
2018
- 2018-11-19 CO CONC2018/0012433A patent/CO2018012433A2/es unknown
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