JP6775584B2 - 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 - Google Patents

設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 Download PDF

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JP6775584B2
JP6775584B2 JP2018531315A JP2018531315A JP6775584B2 JP 6775584 B2 JP6775584 B2 JP 6775584B2 JP 2018531315 A JP2018531315 A JP 2018531315A JP 2018531315 A JP2018531315 A JP 2018531315A JP 6775584 B2 JP6775584 B2 JP 6775584B2
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meganuclease
rho1
residues
rho
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JP2018531624A (ja
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バートセビチ，ビクター
ジャンツ，デレック
ジェファーソン スミス，ジェイムス
ジェファーソン スミス，ジェイムス
ジー． ニコルソン，マイケル
ジー． ニコルソン，マイケル
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プレシジョン バイオサイエンシズ，インク．
プレシジョン バイオサイエンシズ，インク．
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    • A—HUMAN NECESSITIES
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2018531315A 2015-09-08 2016-09-08 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療 Active JP6775584B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562215460P 2015-09-08 2015-09-08
US62/215,460 2015-09-08
PCT/US2016/050809 WO2017044649A1 (en) 2015-09-08 2016-09-08 Treatment of retinitis pigmentosa using engineered meganucleases

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JP2020168987A Division JP7068413B2 (ja) 2015-09-08 2020-10-06 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療

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JP2018531624A JP2018531624A (ja) 2018-11-01
JP2018531624A5 JP2018531624A5 (enExample) 2020-10-01
JP6775584B2 true JP6775584B2 (ja) 2020-10-28

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JP2018531315A Active JP6775584B2 (ja) 2015-09-08 2016-09-08 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療
JP2020168987A Active JP7068413B2 (ja) 2015-09-08 2020-10-06 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療
JP2022074109A Pending JP2022110004A (ja) 2015-09-08 2022-04-28 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療
JP2023182410A Pending JP2024023183A (ja) 2015-09-08 2023-10-24 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療
JP2025079382A Pending JP2025131592A (ja) 2015-09-08 2025-05-12 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療

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JP2020168987A Active JP7068413B2 (ja) 2015-09-08 2020-10-06 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療
JP2022074109A Pending JP2022110004A (ja) 2015-09-08 2022-04-28 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療
JP2023182410A Pending JP2024023183A (ja) 2015-09-08 2023-10-24 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療
JP2025079382A Pending JP2025131592A (ja) 2015-09-08 2025-05-12 設計されたメガヌクレアーゼを使用した網膜色素変性症の治療

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US (6) US10603363B2 (enExample)
EP (2) EP4530354A3 (enExample)
JP (5) JP6775584B2 (enExample)
AU (3) AU2016321244B2 (enExample)
CA (1) CA2997909A1 (enExample)
WO (1) WO2017044649A1 (enExample)

Families Citing this family (13)

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WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
EP3126495A1 (en) 2014-04-02 2017-02-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
US11180793B2 (en) 2015-04-24 2021-11-23 Editas Medicine, Inc. Evaluation of Cas9 molecule/guide RNA molecule complexes
CA2997909A1 (en) * 2015-09-08 2017-03-16 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
KR20230170125A (ko) * 2017-04-21 2023-12-18 프리시젼 바이오사이언시스 인코포레이티드 Pcsk9 유전자 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
AU2018372763A1 (en) * 2017-11-21 2020-05-28 Bayer Healthcare Llc Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa
CN120843482A (zh) 2019-05-07 2025-10-28 精密生物科学公司 用于识别序列的工程化大范围核酸酶的优化
WO2021113763A1 (en) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
WO2021212686A1 (zh) * 2020-04-21 2021-10-28 北京中因科技有限公司 RHO-adRP基于基因编辑的方法和组合物
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
WO2021231495A1 (en) * 2020-05-12 2021-11-18 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using improved engineered meganucleases
US20250228973A1 (en) * 2021-10-19 2025-07-17 Precision Biosciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
WO2025101946A1 (en) 2023-11-08 2025-05-15 Precision Biosciences, Inc. Polypeptide linkers for use in engineered meganucleases

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US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
US6015832A (en) 1997-12-31 2000-01-18 The Regents Of The University Of Michigan Methods of inactivating bacteria including bacterial spores
US6506803B1 (en) 1999-04-28 2003-01-14 Regents Of The University Of Michigan Methods of preventing and treating microbial infections
US6559189B2 (en) 1999-04-28 2003-05-06 Regents Of The University Of Michigan Non-toxic antimicrobial compositions and methods of use
US6635676B2 (en) 1999-04-28 2003-10-21 Regents Of The University Of Michigan Non-toxic antimicrobial compositions and methods of use
US7767216B2 (en) 1999-04-28 2010-08-03 The Regents Of The University Of Michigan Antimicrobial compositions and methods of use
EP2628794B1 (en) 2005-10-18 2016-05-04 Precision Biosciences Rationally designed meganucleases with altered sequence specificity and DNA-binding affinity
WO2009059195A2 (en) 2007-10-31 2009-05-07 Precision Biosciences Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
EP2531609A1 (en) 2010-02-02 2012-12-12 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the diagnosis and therapy of retinitis pigmentosa
EP2569435A1 (en) 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the rhodopsin gene and uses thereof
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AU2016321244A1 (en) 2018-04-12
JP2024023183A (ja) 2024-02-21
JP2018531624A (ja) 2018-11-01
EP3347463C0 (en) 2024-11-13
EP4530354A2 (en) 2025-04-02
AU2016321244B2 (en) 2020-09-24
US20180250370A1 (en) 2018-09-06
US20210000928A1 (en) 2021-01-07
AU2020294264A1 (en) 2021-01-28
US20250121039A1 (en) 2025-04-17
EP4530354A3 (en) 2025-09-10
JP2021054824A (ja) 2021-04-08
US10603363B2 (en) 2020-03-31
US20240238387A1 (en) 2024-07-18
AU2023210619A1 (en) 2023-08-24
EP3347463B1 (en) 2024-11-13
CA2997909A1 (en) 2017-03-16
JP2025131592A (ja) 2025-09-09
JP7068413B2 (ja) 2022-05-16
US20200179495A1 (en) 2020-06-11
EP3347463A1 (en) 2018-07-18
JP2022110004A (ja) 2022-07-28
US10758595B2 (en) 2020-09-01
WO2017044649A1 (en) 2017-03-16
US20220143155A1 (en) 2022-05-12

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