JP2023544633A - 治療における使用のためのｒａｇ１の置き換え - Google Patents

治療における使用のためのｒａｇ１の置き換え Download PDF

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JP2023544633A
JP2023544633A JP2023521740A JP2023521740A JP2023544633A JP 2023544633 A JP2023544633 A JP 2023544633A JP 2023521740 A JP2023521740 A JP 2023521740A JP 2023521740 A JP2023521740 A JP 2023521740A JP 2023544633 A JP2023544633 A JP 2023544633A
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ヴィラ，アンナ
ジェノベーゼ，ピエトロ
ナルディーニ，ルイージ
サケッティ，ニコロ
カルミナ カスティエロ，マリア
フェラーリ，サムエレ
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オスペダーレ サン ラファエレ エス．アール．エル
フォンダツィオーネ テレトン イーティーエス
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2023521740A 2020-10-12 2021-10-12 治療における使用のためのｒａｇ１の置き換え Pending JP2023544633A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB2016139.4A GB202016139D0 (en) 2020-10-12 2020-10-12 Polynucleotide
GB2016139.4 2020-10-12
AU2021202657A AU2021202657A1 (en) 2021-04-28 2021-04-28 Polynucleotide
AU2021202657 2021-04-28
PCT/EP2021/078222 WO2022079054A1 (en) 2020-10-12 2021-10-12 Replacement of rag1 for use in therapy

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JP2023544633A true JP2023544633A (ja) 2023-10-24
JP2023544633A5 JP2023544633A5 (https=) 2024-10-22

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JP2023521740A Pending JP2023544633A (ja) 2020-10-12 2021-10-12 治療における使用のためのｒａｇ１の置き換え

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US (1) US20230365996A1 (https=)
EP (1) EP4225900A1 (https=)
JP (1) JP2023544633A (https=)
AU (1) AU2021359781A1 (https=)
IL (1) IL302031A (https=)
WO (1) WO2022079054A1 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024538769A (ja) * 2021-10-12 2024-10-23 オスペダーレ サン ラファエレ エス．アール．エル Ｒａｇ１遺伝子における突然変異を修正するのに有用なポリヌクレオチド
GB202206346D0 (en) * 2022-04-29 2022-06-15 Ospedale San Raffaele Srl Gene therapy
WO2025073293A1 (en) * 2023-10-02 2025-04-10 Consorcio Centro De Investigación Biomédica En Red Gene editing methods for cd34+ cells to achieve clinically relevant efficacies

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190038771A1 (en) * 2016-02-02 2019-02-07 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome

Family Cites Families (2)

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Publication number Priority date Publication date Assignee Title
PT904392E (pt) 1996-10-17 2001-06-29 Oxford Biomedica Ltd Vectores retrovirais
AU2019294294A1 (en) 2018-06-25 2021-02-11 Fondazione Telethon Ets Gene therapy

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190038771A1 (en) * 2016-02-02 2019-02-07 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
ESGCT XXV ANNIVERSARY CONGRESS IN COLLABORATION WITH THE GERMAN SOCIETY FOR GENE THERAPY, OCTOBER 17, vol. A8, JPN6026010727, 2017, ISSN: 0005822409 *
J CLIN IMMUNOL., vol. Vol.34, Suppl. 2, JPN6026010728, 2014, pages 183, ISSN: 0005822408 *
NATURE COMMUNICATIONS, vol. 11, JPN6026010726, 12 August 2020 (2020-08-12), pages 1 - 15, ISSN: 0005822410 *
SCI. ADV., vol. 6, JPN6026010725, 6 May 2020 (2020-05-06), pages 0571 - 1, ISSN: 0005822411 *

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EP4225900A1 (en) 2023-08-16
US20230365996A1 (en) 2023-11-16
IL302031A (en) 2023-06-01
AU2021359781A1 (en) 2023-06-15
WO2022079054A1 (en) 2022-04-21
AU2021359781A9 (en) 2025-04-10

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