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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JP2018531624A5 (OSRAM
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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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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • 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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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • 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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  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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 (OSRAM) 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 (OSRAM)
EP (2) EP3347463B1 (OSRAM)
JP (5) JP6775584B2 (OSRAM)
AU (3) AU2016321244B2 (OSRAM)
CA (1) CA2997909A1 (OSRAM)
WO (1) WO2017044649A1 (OSRAM)

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EP3114227B1 (en) 2014-03-05 2021-07-21 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
WO2016172727A1 (en) 2015-04-24 2016-10-27 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
EP3347463B1 (en) * 2015-09-08 2024-11-13 Precision Biosciences, Inc. Treatment of retinitis pigmentosa using engineered meganucleases
US11680254B2 (en) * 2017-04-21 2023-06-20 Precision Biosciences, Inc. Engineered meganucleases specific for recognition sequences in the PCSK9 gene
MA50877A (fr) * 2017-11-21 2020-09-30 Bayer Healthcare Llc Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante
CA3137975A1 (en) 2019-05-07 2020-11-12 Precision Biosciences, Inc. Optimization of engineered meganucleases for recognition sequences
US20230033866A1 (en) * 2019-12-06 2023-02-02 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
EP4419660A2 (en) * 2021-10-19 2024-08-28 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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US20130071373A1 (en) * 2010-02-02 2013-03-21 Christina Zeitz Methods for the diagnosis and therapy of retinitis pigmentosa
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US10603363B2 (en) 2020-03-31
US20200179495A1 (en) 2020-06-11
EP3347463A1 (en) 2018-07-18
EP3347463B1 (en) 2024-11-13
US20180250370A1 (en) 2018-09-06
AU2020294264A1 (en) 2021-01-28
WO2017044649A1 (en) 2017-03-16
JP7068413B2 (ja) 2022-05-16
AU2023210619A1 (en) 2023-08-24
US20250121039A1 (en) 2025-04-17
EP4530354A2 (en) 2025-04-02
AU2016321244B2 (en) 2020-09-24
JP2021054824A (ja) 2021-04-08
EP3347463C0 (en) 2024-11-13
JP2018531624A (ja) 2018-11-01
US20210000928A1 (en) 2021-01-07
CA2997909A1 (en) 2017-03-16
AU2016321244A1 (en) 2018-04-12
JP2022110004A (ja) 2022-07-28
US20240238387A1 (en) 2024-07-18
US20220143155A1 (en) 2022-05-12
US10758595B2 (en) 2020-09-01
JP2024023183A (ja) 2024-02-21
JP2025131592A (ja) 2025-09-09
EP4530354A3 (en) 2025-09-10

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