JPWO2020176552A5 - - Google Patents
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- Publication number
- JPWO2020176552A5 JPWO2020176552A5 JP2021549698A JP2021549698A JPWO2020176552A5 JP WO2020176552 A5 JPWO2020176552 A5 JP WO2020176552A5 JP 2021549698 A JP2021549698 A JP 2021549698A JP 2021549698 A JP2021549698 A JP 2021549698A JP WO2020176552 A5 JPWO2020176552 A5 JP WO2020176552A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- molecule
- grna
- rho
- grna molecule
- 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
- 108020004707 nucleic acids Proteins 0.000 claims description 117
- 150000007523 nucleic acids Chemical class 0.000 claims description 117
- 102000039446 nucleic acids Human genes 0.000 claims description 117
- 108020005004 Guide RNA Proteins 0.000 claims description 67
- 238000000034 method Methods 0.000 claims description 38
- 101150079354 rho gene Proteins 0.000 claims description 24
- 230000008685 targeting Effects 0.000 claims description 24
- 239000002773 nucleotide Substances 0.000 claims description 19
- 125000003729 nucleotide group Chemical group 0.000 claims description 19
- 101710163270 Nuclease Proteins 0.000 claims description 18
- 239000002299 complementary DNA Substances 0.000 claims description 18
- 230000000295 complement effect Effects 0.000 claims description 11
- 239000013607 AAV vector Substances 0.000 claims description 10
- 239000000203 mixture Substances 0.000 claims description 8
- 108091033409 CRISPR Proteins 0.000 claims description 5
- 230000002207 retinal effect Effects 0.000 claims description 3
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical compound O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims 3
- 102000004330 Rhodopsin Human genes 0.000 claims 3
- 108090000820 Rhodopsin Proteins 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 102100040756 Rhodopsin Human genes 0.000 description 27
- 210000004027 cell Anatomy 0.000 description 22
- 108020005345 3' Untranslated Regions Proteins 0.000 description 10
- 230000035772 mutation Effects 0.000 description 7
- 230000005783 single-strand break Effects 0.000 description 6
- 230000005782 double-strand break Effects 0.000 description 4
- 238000003776 cleavage reaction Methods 0.000 description 3
- 230000007017 scission Effects 0.000 description 3
- 101100518359 Homo sapiens RHO gene Proteins 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- 238000012163 sequencing technique Methods 0.000 description 2
- 102100029362 Cone-rod homeobox protein Human genes 0.000 description 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 1
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 1
- 102100024125 Embryonal Fyn-associated substrate Human genes 0.000 description 1
- 102100027685 Hemoglobin subunit alpha Human genes 0.000 description 1
- 108091005902 Hemoglobin subunit alpha Proteins 0.000 description 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 description 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 description 1
- 101000919370 Homo sapiens Cone-rod homeobox protein Proteins 0.000 description 1
- 101001053896 Homo sapiens Embryonal Fyn-associated substrate Proteins 0.000 description 1
- 101000710137 Homo sapiens Recoverin Proteins 0.000 description 1
- 101000829506 Homo sapiens Rhodopsin kinase GRK1 Proteins 0.000 description 1
- 102100034268 Neural retina-specific leucine zipper protein Human genes 0.000 description 1
- 101710181914 Neural retina-specific leucine zipper protein Proteins 0.000 description 1
- 102100034572 Recoverin Human genes 0.000 description 1
- 102100023742 Rhodopsin kinase GRK1 Human genes 0.000 description 1
- 210000001671 embryonic stem cell Anatomy 0.000 description 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 description 1
- 238000009396 hybridization Methods 0.000 description 1
- 210000004263 induced pluripotent stem cell Anatomy 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 210000002901 mesenchymal stem cell Anatomy 0.000 description 1
- 230000001537 neural effect Effects 0.000 description 1
- 239000011541 reaction mixture Substances 0.000 description 1
- 210000000880 retinal rod photoreceptor cell Anatomy 0.000 description 1
- 210000000130 stem cell Anatomy 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962810320P | 2019-02-25 | 2019-02-25 | |
| US62/810,320 | 2019-02-25 | ||
| PCT/US2020/019766 WO2020176552A1 (en) | 2019-02-25 | 2020-02-25 | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022521764A JP2022521764A (ja) | 2022-04-12 |
| JPWO2020176552A5 true JPWO2020176552A5 (enExample) | 2023-03-07 |
Family
ID=69960739
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021549698A Pending JP2022521764A (ja) | 2019-02-25 | 2020-02-25 | Ｒｈｏ関連常染色体優性網膜色素変性症（ａｄｒｐ）を治療するためのｃｒｉｓｐｒ／ｒｎａ誘導ヌクレアーゼ関連の方法及び組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220133768A1 (enExample) |
| EP (1) | EP3931326A1 (enExample) |
| JP (1) | JP2022521764A (enExample) |
| KR (1) | KR20210133993A (enExample) |
| CN (1) | CN113631710A (enExample) |
| AU (1) | AU2020227740A1 (enExample) |
| CA (1) | CA3130515A1 (enExample) |
| IL (1) | IL285680A (enExample) |
| WO (1) | WO2020176552A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220112516A1 (en) * | 2018-11-08 | 2022-04-14 | National University Corporation Tokai National Higher Education And Research System | Gene therapy employing genome editing with single aav vector |
| US20230054569A1 (en) * | 2019-12-18 | 2023-02-23 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| US20230399638A1 (en) * | 2020-11-01 | 2023-12-14 | University of South Alabama Foundation for Research and Commercialization | Barcoded Cells Engineered With Heterozygous Genetic Diversity |
| US20240207448A1 (en) * | 2021-04-16 | 2024-06-27 | Editas Medicine, Inc. | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
| WO2023285431A1 (en) * | 2021-07-12 | 2023-01-19 | Alia Therapeutics Srl | Compositions and methods for allele specific treatment of retinitis pigmentosa |
| WO2024036366A1 (en) * | 2022-08-16 | 2024-02-22 | The University Of Adelaide | Agent for treating or preventing a dominantly-inherited disease |
| CA3267752A1 (en) * | 2022-09-16 | 2024-03-21 | Alia Therapeutics Srl | ENQP TYPE II CAS PROTEINS AND THEIR APPLICATIONS |
| EP4590819A2 (en) * | 2022-09-20 | 2025-07-30 | The Board of Trustees of the Leland Stanford Junior University | Methods and compositions to control gene using genome editing |
| CN116555259B (zh) * | 2023-04-20 | 2024-02-27 | 云舟生物科技(广州)股份有限公司 | 核酸分子及其作为启动子的应用 |
| WO2025090637A2 (en) * | 2023-10-23 | 2025-05-01 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of retinitis pigmentosa |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5443505A (en) | 1993-11-15 | 1995-08-22 | Oculex Pharmaceuticals, Inc. | Biocompatible ocular implants |
| US5725493A (en) | 1994-12-12 | 1998-03-10 | Avery; Robert Logan | Intravitreal medicine delivery |
| US6299895B1 (en) | 1997-03-24 | 2001-10-09 | Neurotech S.A. | Device and method for treating ophthalmic diseases |
| KR100732262B1 (ko) | 1999-10-21 | 2007-06-25 | 알콘, 인코퍼레이티드 | 약물 전달 장치 |
| US6416777B1 (en) | 1999-10-21 | 2002-07-09 | Alcon Universal Ltd. | Ophthalmic drug delivery device |
| WO2015089462A1 (en) * | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| EP3114227B1 (en) | 2014-03-05 | 2021-07-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| US9970001B2 (en) * | 2014-06-05 | 2018-05-15 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease design |
| AU2015277369B2 (en) * | 2014-06-16 | 2021-08-19 | The Johns Hopkins University | Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter |
| US20160324987A1 (en) * | 2015-04-15 | 2016-11-10 | Cedars-Sinai Medical Center | Use of crispr/cas9 as in vivo gene therapy to generate targeted genomic disruptions in genes bearing dominant mutations for retinitis pigmentosa |
| EP4008780A1 (en) * | 2015-04-30 | 2022-06-08 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| US11512311B2 (en) | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
| CN109890424A (zh) * | 2016-07-05 | 2019-06-14 | 约翰霍普金斯大学 | 用于治疗视网膜变性的基于crispr/cas9的组合物和方法 |
| KR102604903B1 (ko) | 2016-12-05 | 2023-11-21 | 에디타스 메디신, 인코포레이티드 | 내인성 및 공급원 DNA의 원-샷 가이드 RNA(ogRNA) 표적화를 위한 시스템 및 방법 |
| EP3622070A2 (en) * | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| EP3714055A1 (en) * | 2017-11-21 | 2020-09-30 | CRISPR Therapeutics AG | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
| EP3768327A4 (en) * | 2018-03-23 | 2022-04-13 | The Trustees of Columbia University in the City of New York | GENE EDITING FOR AUTOSOMAL DOMINANT DISEASES |
-
2020
- 2020-02-25 JP JP2021549698A patent/JP2022521764A/ja active Pending
- 2020-02-25 CA CA3130515A patent/CA3130515A1/en active Pending
- 2020-02-25 AU AU2020227740A patent/AU2020227740A1/en not_active Abandoned
- 2020-02-25 KR KR1020217030652A patent/KR20210133993A/ko active Pending
- 2020-02-25 EP EP20714062.5A patent/EP3931326A1/en active Pending
- 2020-02-25 WO PCT/US2020/019766 patent/WO2020176552A1/en not_active Ceased
- 2020-02-25 US US17/433,975 patent/US20220133768A1/en active Pending
- 2020-02-25 CN CN202080025160.5A patent/CN113631710A/zh active Pending
-
2021
- 2021-08-17 IL IL285680A patent/IL285680A/en unknown
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