CN113631710A - 用于治疗rho相关的常染色体显性视网膜色素变性(adrp)的crispr/rna指导的核酸酶相关方法和组合物 - Google Patents

用于治疗rho相关的常染色体显性视网膜色素变性(adrp)的crispr/rna指导的核酸酶相关方法和组合物 Download PDF

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CN113631710A
CN113631710A CN202080025160.5A CN202080025160A CN113631710A CN 113631710 A CN113631710 A CN 113631710A CN 202080025160 A CN202080025160 A CN 202080025160A CN 113631710 A CN113631710 A CN 113631710A
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nucleic acid
rho
molecule
grna
rna
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B·A·戴纳
D·雷昂
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Editas Medicine Inc
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Editas Medicine Inc
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CN202080025160.5A 2019-02-25 2020-02-25 用于治疗rho相关的常染色体显性视网膜色素变性(adrp)的crispr/rna指导的核酸酶相关方法和组合物 Pending CN113631710A (zh)

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)

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Publication Number Publication Date
CN113631710A true CN113631710A (zh) 2021-11-09

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CN202080025160.5A Pending CN113631710A (zh) 2019-02-25 2020-02-25 用于治疗rho相关的常染色体显性视网膜色素变性(adrp)的crispr/rna指导的核酸酶相关方法和组合物

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US (1) US20220133768A1 (https=)
EP (1) EP3931326A1 (https=)
JP (1) JP2022521764A (https=)
KR (1) KR20210133993A (https=)
CN (1) CN113631710A (https=)
AU (1) AU2020227740A1 (https=)
CA (1) CA3130515A1 (https=)
IL (1) IL285680A (https=)
WO (1) WO2020176552A1 (https=)

Cited By (1)

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WO2024217518A1 (zh) * 2023-04-20 2024-10-24 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

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WO2020096049A1 (ja) * 2018-11-08 2020-05-14 国立大学法人東北大学 単一のａａｖベクターによるゲノム編集を用いた遺伝子治療
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
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
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
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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WO2024217518A1 (zh) * 2023-04-20 2024-10-24 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

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AU2020227740A1 (en) 2021-10-07
KR20210133993A (ko) 2021-11-08
EP3931326A1 (en) 2022-01-05
US20220133768A1 (en) 2022-05-05
JP2022521764A (ja) 2022-04-12
IL285680A (en) 2021-10-31
CA3130515A1 (en) 2020-09-03
WO2020176552A1 (en) 2020-09-03

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