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
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 (https=) | 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 (https=) |
| EP (1) | EP3445375A1 (https=) |
| JP (1) | JP2019515914A (https=) |
| KR (1) | KR20180134412A (https=) |
| CN (1) | CN109414450A (https=) |
| AU (1) | AU2017254718A1 (https=) |
| BR (1) | BR112018071439A2 (https=) |
| CA (1) | CA3021647A1 (https=) |
| CO (1) | CO2018012433A2 (https=) |
| MX (1) | MX2018012873A (https=) |
| WO (1) | WO2017185054A1 (https=) |
Families Citing this family (20)
| 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 |
| TWI851534B (zh) | 2016-12-22 | 2024-08-11 | 美商英特利亞醫療公司 | 用於治療α-１抗胰蛋白酶缺乏症之組合物及方法 |
| WO2018165541A1 (en) | 2017-03-10 | 2018-09-13 | The Board Of Regents Of The University Of Texas System | Treatment of fuchs' endothelial corneal dystrophy |
| WO2019118875A1 (en) * | 2017-12-15 | 2019-06-20 | Ou Li | 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 |
| EP3728598A1 (en) * | 2017-12-21 | 2020-10-28 | 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 |
| CA3129856A1 (en) * | 2019-02-28 | 2020-09-03 | Friedrich E. Kruse | Extracellular matrix modulating agent |
| US20220047618A1 (en) * | 2019-02-28 | 2022-02-17 | Regeneron Pharmaceuticals, Inc. | Adeno-associated virus vectors for the delivery of therapeutics |
| EP4126224A4 (en) * | 2020-04-27 | 2024-07-03 | Duke University | HIGH-THROUGHPUT SCREENING METHODS TO DISCOVER OPTIMAL GRNA PAIRS FOR CRISPR-MEDIATED EXON DELETION |
| 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 |
| 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 |
| IL303506A (en) | 2020-12-11 | 2023-08-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing involving deamination |
| US12171813B2 (en) | 2021-02-05 | 2024-12-24 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| EP4430185A1 (en) * | 2021-11-09 | 2024-09-18 | 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 |
| WO2024102677A1 (en) | 2022-11-08 | 2024-05-16 | Orna Therapeutics, Inc. | Circular rna compositions |
| 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 |
|---|---|---|---|---|
| US20060051405A1 (en) | 2004-07-19 | 2006-03-09 | Protiva Biotherapeutics, Inc. | Compositions for the delivery of therapeutic agents and uses thereof |
| WO2013101711A1 (en) * | 2011-12-30 | 2013-07-04 | Mayo Foundation For Medical Education And Research | Assessing likelihood of developing fuchs' corneal dystrophy |
| 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 |
| 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 |
| CN107879960B (zh) | 2013-03-08 | 2021-06-22 | 诺华股份有限公司 | 用于传递活性成分的脂质和脂质组合物 |
| CN106459995B (zh) * | 2013-11-07 | 2020-02-21 | 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 |
| EP3470089A1 (en) * | 2013-12-12 | 2019-04-17 | 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 |
| WO2015161276A2 (en) * | 2014-04-18 | 2015-10-22 | 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 KR KR1020187033525A patent/KR20180134412A/ko not_active Ceased
- 2017-04-21 EP EP17722552.1A patent/EP3445375A1/en not_active Withdrawn
- 2017-04-21 JP JP2018555262A patent/JP2019515914A/ja active Pending
- 2017-04-21 WO PCT/US2017/028981 patent/WO2017185054A1/en not_active Ceased
- 2017-04-21 CA CA3021647A patent/CA3021647A1/en active Pending
- 2017-04-21 US US16/095,236 patent/US20190142972A1/en not_active Abandoned
- 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 CN CN201780036135.5A patent/CN109414450A/zh active Pending
- 2017-04-21 MX MX2018012873A patent/MX2018012873A/es unknown
-
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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