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
- 108020005004 Guide RNA Proteins 0.000 claims description 195
- 101710163270 Nuclease Proteins 0.000 claims description 182
- 239000000203 mixture Substances 0.000 claims description 99
- 108090000623 proteins and genes Proteins 0.000 claims description 31
- 239000002773 nucleotide Substances 0.000 claims description 18
- 125000003729 nucleotide group Chemical group 0.000 claims description 18
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 16
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 16
- 230000000295 complement effect 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
- 102220055955 rs727504229 Human genes 0.000 claims description 9
- 102200030577 rs80358191 Human genes 0.000 claims description 9
- 102000009523 Transcription Factor 4 Human genes 0.000 claims description 8
- 108010048992 Transcription Factor 4 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
- 230000006641 stabilisation Effects 0.000 claims description 7
- 238000011105 stabilization Methods 0.000 claims description 7
- 239000013598 vector Substances 0.000 claims description 7
- 108700028369 Alleles Proteins 0.000 claims description 4
- 101150030360 COL8A2 gene Proteins 0.000 claims description 4
- 101100114043 Homo sapiens COL8A2 gene Proteins 0.000 claims description 4
- 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
- 208000003923 Hereditary Corneal Dystrophies Diseases 0.000 claims description 3
- 238000011260 co-administration Methods 0.000 claims description 3
- 206010011005 corneal dystrophy Diseases 0.000 claims description 3
- 239000003814 drug Substances 0.000 claims description 3
- 239000003889 eye drop Substances 0.000 claims description 3
- 229940012356 eye drops Drugs 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
- 208000010157 sclerosing cholangitis Diseases 0.000 claims description 3
- 239000013603 viral vector Substances 0.000 claims description 3
- 108091033409 CRISPR Proteins 0.000 claims description 2
- 238000010453 CRISPR/Cas method Methods 0.000 claims description 2
- 108010069526 Collagen Type VIII Proteins 0.000 claims description 2
- 102000001191 Collagen Type VIII Human genes 0.000 claims description 2
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 2
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 230000009977 dual effect Effects 0.000 claims description 2
- 238000009472 formulation Methods 0.000 claims 8
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 102000040945 Transcription factor Human genes 0.000 claims 1
- 108091023040 Transcription factor Proteins 0.000 claims 1
- 230000004452 decreased vision Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 33
- 102100040496 Collagen alpha-2(VIII) chain Human genes 0.000 description 4
- 101000749886 Homo sapiens Collagen alpha-2(VIII) chain Proteins 0.000 description 4
- 230000007423 decrease Effects 0.000 description 2
- 101150017815 TCF4 gene Proteins 0.000 description 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 (cg-RX-API-DMAC7.html) | 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)
| Country | Link |
|---|---|
| US (2) | US20190142972A1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3445375A1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2019515914A (cg-RX-API-DMAC7.html) |
| KR (1) | KR20180134412A (cg-RX-API-DMAC7.html) |
| CN (1) | CN109414450A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2017254718A1 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112018071439A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3021647A1 (cg-RX-API-DMAC7.html) |
| CO (1) | CO2018012433A2 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2018012873A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2017185054A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2989331A1 (en) * | 2015-07-02 | 2017-01-05 | The Johns Hopkins University | Crispr/cas9-based treatments |
| 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 |
| KR102551664B1 (ko) | 2016-12-22 | 2023-07-05 | 인텔리아 테라퓨틱스, 인크. | 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법 |
| WO2018165541A1 (en) | 2017-03-10 | 2018-09-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
| EP3723813A4 (en) * | 2017-12-15 | 2021-10-06 | Regents of the University of Minnesota | GENOMIC CRISPR MEDIATION 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 |
| WO2019122302A1 (en) | 2017-12-21 | 2019-06-27 | 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 |
| TW202045186A (zh) * | 2019-02-28 | 2020-12-16 | 弗里德里希 Ｅ 克魯斯 | 細胞外基質調節劑 |
| JP2022522756A (ja) * | 2019-02-28 | 2022-04-20 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 治療薬の送達のためのアデノ随伴ウイルスベクター |
| WO2021222327A1 (en) * | 2020-04-27 | 2021-11-04 | Duke University | A high-throughput screening method to discover optimal grna pairs for crispr-mediated exon deletion |
| 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 |
| JP2023553935A (ja) | 2020-12-11 | 2023-12-26 | インテリア セラピューティクス，インコーポレイテッド | 脱アミノ化を伴うゲノム編集のためのポリヌクレオチド、組成物、及び方法 |
| EP4288541A1 (en) | 2021-02-05 | 2023-12-13 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| WO2023086842A1 (en) * | 2021-11-09 | 2023-05-19 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy |
| US12297285B2 (en) | 2022-06-24 | 2025-05-13 | Orna Therapeutics, Inc. | Circular RNA encoding chimeric antigen receptors targeting BCMA |
| WO2024226536A1 (en) * | 2023-04-24 | 2024-10-31 | The General Hospital Corporation | Methods and compositions for modifying genetic repeats |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
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 |
| GB2537000C (en) * | 2012-05-25 | 2019-10-09 | Univ California | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| EP2931899A1 (en) * | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| KR102255108B1 (ko) | 2013-03-08 | 2021-05-24 | 노파르티스 아게 | 활성제의 전달을 위한 지질 및 지질 조성물 |
| CN111218447B (zh) * | 2013-11-07 | 2024-10-11 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| WO2015089419A2 (en) * | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| KR102595473B1 (ko) * | 2014-04-18 | 2023-10-30 | 에디타스 메디신, 인코포레이티드 | 암 면역요법을 위한 crispr-cas-관련 방법, 조성물 및 구성성분 |
| CA2989331A1 (en) * | 2015-07-02 | 2017-01-05 | The Johns Hopkins University | Crispr/cas9-based treatments |
-
2017
- 2017-04-21 BR BR112018071439-9A patent/BR112018071439A2/pt not_active IP Right Cessation
- 2017-04-21 WO PCT/US2017/028981 patent/WO2017185054A1/en not_active Ceased
- 2017-04-21 CN CN201780036135.5A patent/CN109414450A/zh active Pending
- 2017-04-21 MX MX2018012873A patent/MX2018012873A/es unknown
- 2017-04-21 KR KR1020187033525A patent/KR20180134412A/ko not_active Ceased
- 2017-04-21 US US16/095,236 patent/US20190142972A1/en not_active Abandoned
- 2017-04-21 EP EP17722552.1A patent/EP3445375A1/en not_active Withdrawn
- 2017-04-21 AU AU2017254718A patent/AU2017254718A1/en not_active Abandoned
- 2017-04-21 CA CA3021647A patent/CA3021647A1/en active Pending
- 2017-04-21 JP JP2018555262A patent/JP2019515914A/ja active Pending
-
2018
- 2018-11-19 CO CONC2018/0012433A patent/CO2018012433A2/es unknown
-
2024
- 2024-01-17 US US18/415,032 patent/US20250025580A1/en active Pending
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