JP2022521764A - 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 - Google Patents

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 Download PDF

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JP2022521764A
JP2022521764A JP2021549698A JP2021549698A JP2022521764A JP 2022521764 A JP2022521764 A JP 2022521764A JP 2021549698 A JP2021549698 A JP 2021549698A JP 2021549698 A JP2021549698 A JP 2021549698A JP 2022521764 A JP2022521764 A JP 2022521764A
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ダイナー、ベンジャミン・アリー
レヨン、ディーパック
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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)

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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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JP2022521764A true JP2022521764A (ja) 2022-04-12
JPWO2020176552A5 JPWO2020176552A5 (https=) 2023-03-07
JP2022521764A5 JP2022521764A5 (https=) 2023-03-07

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CN (1) CN113631710A (https=)
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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
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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017504312A (ja) * 2013-12-12 2017-02-09 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ−Ｃａｓ系及び組成物の送達、使用及び治療適用
JP2017519500A (ja) * 2014-06-16 2017-07-20 ザ・ジョンズ・ホプキンス・ユニバーシティー Ｈ１プロモーターを用いるｃｒｉｓｐｒガイドｒｎａの発現のための組成物および方法
WO2018009562A1 (en) * 2016-07-05 2018-01-11 The Johns Hopkins University Crispr/cas9-based compositions and methods for treating retinal degenerations
WO2018209158A2 (en) * 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods

Family Cites Families (13)

* 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
ATE283013T1 (de) 1999-10-21 2004-12-15 Alcon Inc Medikamentenzuführvorrichtung
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
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
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
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

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017504312A (ja) * 2013-12-12 2017-02-09 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ−Ｃａｓ系及び組成物の送達、使用及び治療適用
JP2017519500A (ja) * 2014-06-16 2017-07-20 ザ・ジョンズ・ホプキンス・ユニバーシティー Ｈ１プロモーターを用いるｃｒｉｓｐｒガイドｒｎａの発現のための組成物および方法
WO2018009562A1 (en) * 2016-07-05 2018-01-11 The Johns Hopkins University Crispr/cas9-based compositions and methods for treating retinal degenerations
WO2018209158A2 (en) * 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BURNIGHT, ERIN R. ET AL., MOLECULAR THERAPY, vol. Vol. 25, Issue 9, JPN6024001092, 6 September 2017 (2017-09-06), pages 1999 - 2013, ISSN: 0005238464 *
TSAI, YI-TING ET AL., OPHTHALMOLOGY, vol. Vol. 125, Issue 9, JPN6024001091, September 2018 (2018-09-01), pages 1421 - 1430, ISSN: 0005442376 *

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KR20210133993A (ko) 2021-11-08
EP3931326A1 (en) 2022-01-05
US20220133768A1 (en) 2022-05-05
IL285680A (en) 2021-10-31
CN113631710A (zh) 2021-11-09
CA3130515A1 (en) 2020-09-03
WO2020176552A1 (en) 2020-09-03

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