KR20210133993A - Rho-연관 상염색체-우성 망막색소변성증(adrp)을 치료하기 위한 crispr/rna-가이드 뉴클레아제-관련 방법 및 조성물 - Google Patents
Rho-연관 상염색체-우성 망막색소변성증(adrp)을 치료하기 위한 crispr/rna-가이드 뉴클레아제-관련 방법 및 조성물 Download PDFInfo
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| 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) |
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| WO2021122944A1 (en) * | 2019-12-18 | 2021-06-24 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| WO2022094363A1 (en) * | 2020-11-01 | 2022-05-05 | University of South Alabama Foundation for Research and Commercialization | Barcoded cells engineered with heterozygous genetic diversity |
| EP4323522A1 (en) * | 2021-04-16 | 2024-02-21 | 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 |
| US20260048074A1 (en) * | 2022-08-16 | 2026-02-19 | The University Of Adelaide | Agent for treating or preventing a dominantly-inherited disease |
| US20260014279A1 (en) * | 2022-09-16 | 2026-01-15 | Alia Therapeutics Srl | Enqp type cas proteins and applications thereof |
| US20260092271A1 (en) * | 2022-09-20 | 2026-04-02 | 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 |
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| US5443505A (en) | 1993-11-15 | 1995-08-22 | Oculex Pharmaceuticals, Inc. | Biocompatible ocular implants |
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| US6416777B1 (en) | 1999-10-21 | 2002-07-09 | Alcon Universal Ltd. | Ophthalmic drug delivery device |
| ATE283013T1 (de) | 1999-10-21 | 2004-12-15 | Alcon Inc | Medikamentenzuführvorrichtung |
| AU2014361781B2 (en) * | 2013-12-12 | 2021-04-01 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing |
| WO2015134812A1 (en) | 2014-03-05 | 2015-09-11 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| EP3152319A4 (en) * | 2014-06-05 | 2017-12-27 | Sangamo BioSciences, Inc. | Methods and compositions for nuclease design |
| DK3155101T3 (da) * | 2014-06-16 | 2020-05-04 | Univ Johns Hopkins | Sammensætninger og fremgangsmåder til ekspression af CRISPR-leder-RNA´er under anvendelse af H1-promotoren |
| 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 |
| EP3289080B1 (en) * | 2015-04-30 | 2021-08-25 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| BR112019000057A2 (pt) * | 2016-07-05 | 2019-04-02 | The Johns Hopkins University | composições e métodos com base em crispr/cas9 para o tratamento de degeneração de retina |
| WO2018106693A1 (en) | 2016-12-05 | 2018-06-14 | Editas Medicine, Inc. | SYSTEMS AND METHODS FOR ONE-SHOT GUIDE RNA (ogRNA) TARGETING OF ENDOGENOUS AND SOURCE DNA |
| EP3622070A2 (en) * | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| MA50877A (fr) * | 2017-11-21 | 2020-09-30 | Bayer Healthcare Llc | Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante |
| WO2019183630A2 (en) * | 2018-03-23 | 2019-09-26 | The Trustees Of Columbia University In The City Of New York | Gene editing for autosomal dominant diseases |
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