JP2022521764A5 - - Google Patents

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Publication number
JP2022521764A5
JP2022521764A5 JP2021549698A JP2021549698A JP2022521764A5 JP 2022521764 A5 JP2022521764 A5 JP 2022521764A5 JP 2021549698 A JP2021549698 A JP 2021549698A JP 2021549698 A JP2021549698 A JP 2021549698A JP 2022521764 A5 JP2022521764 A5 JP 2022521764A5
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JP
Japan
Prior art keywords
nucleic acid
molecule
grna
rho
acid according
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
Application number
JP2021549698A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022521764A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/019766 external-priority patent/WO2020176552A1/en
Publication of JP2022521764A publication Critical patent/JP2022521764A/ja
Publication of JP2022521764A5 publication Critical patent/JP2022521764A5/ja
Pending legal-status Critical Current

Links

JP2021549698A 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 Pending JP2022521764A (ja)

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
JP2022521764A5 true JP2022521764A5 (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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US20230399638A1 (en) * 2020-11-01 2023-12-14 University of South Alabama Foundation for Research and Commercialization Barcoded Cells Engineered With Heterozygous Genetic Diversity
WO2022221741A1 (en) * 2021-04-16 2022-10-20 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
AU2023326046A1 (en) * 2022-08-16 2025-02-20 Adelaide university Agent for treating or preventing a dominantly-inherited disease
EP4587564A2 (en) * 2022-09-16 2025-07-23 Alia Therapeutics Srl Enqp type ii cas proteins and applications thereof
WO2024064761A2 (en) * 2022-09-20 2024-03-28 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)

* Cited by examiner, † Cited by third party
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
US6416777B1 (en) 1999-10-21 2002-07-09 Alcon Universal Ltd. Ophthalmic drug delivery device
TR200201047T2 (tr) 1999-10-21 2002-07-22 Alcon, Inc. İlaç verme vasıtası.
CN111206032B (zh) * 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
US11028388B2 (en) 2014-03-05 2021-06-08 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
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
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
SG10202109385QA (en) * 2016-07-05 2021-10-28 Univ Johns Hopkins Crispr/cas9-based compositions and methods for treating retinal degenerations
US9963719B1 (en) 2016-12-05 2018-05-08 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
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 EDIT FOR AUTOSOMAL DOMINANT DISEASES

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