JP2023506482A - 遺伝子編集のための修飾されたガイドｒｎａ - Google Patents

遺伝子編集のための修飾されたガイドｒｎａ Download PDF

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JP2023506482A
JP2023506482A JP2022535572A JP2022535572A JP2023506482A JP 2023506482 A JP2023506482 A JP 2023506482A JP 2022535572 A JP2022535572 A JP 2022535572A JP 2022535572 A JP2022535572 A JP 2022535572A JP 2023506482 A JP2023506482 A JP 2023506482A
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grna
nucleotides
modification
positions
region
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アレクサンダー，セス，シー．
ムールパティ，サビン
ロイ，マシュー
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インテリア セラピューティクス，インコーポレイテッド
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/50—Methods for regulating/modulating their activity
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2022535572A 2019-12-11 2020-12-10 遺伝子編集のための修飾されたガイドｒｎａ Pending JP2023506482A (ja)

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US201962946905P 2019-12-11 2019-12-11
US62/946,905 2019-12-11
PCT/US2020/064250 WO2021119275A1 (en) 2019-12-11 2020-12-10 Modified guide rnas for gene editing

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US (1) US20220372483A1 (https=)
EP (1) EP4073249A1 (https=)
JP (2) JP2023506482A (https=)
KR (1) KR20220126725A (https=)
CN (1) CN115176001A (https=)
AU (1) AU2020401206A1 (https=)
BR (1) BR112022011214A2 (https=)
CA (1) CA3164192A1 (https=)
CO (1) CO2022009562A2 (https=)
IL (1) IL293569A (https=)
MX (1) MX2022006950A (https=)
TW (1) TW202136509A (https=)
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CN119384498A (zh) 2022-06-16 2025-01-28 因特利亚治疗公司 用于对细胞进行遗传修饰的方法和组合物
AU2023293131A1 (en) 2022-06-16 2024-12-12 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class i in a cell
WO2024006955A1 (en) 2022-06-29 2024-01-04 Intellia Therapeutics, Inc. Engineered t cells
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TW202436622A (zh) 2023-03-06 2024-09-16 美商英特利亞醫療公司 用於ｂ型肝炎病毒（ｈｂｖ）基因體編輯之組合物及方法
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WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025101994A2 (en) 2023-11-10 2025-05-15 Intellia Therapeutics, Inc. Compositions, methods, and systems for genomic editing
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025137301A1 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells
WO2025137439A2 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Engineered t cells
WO2025184520A1 (en) 2024-02-29 2025-09-04 Intellia Therapeutics, Inc. Compositions and methods for angiopoietin like 3 (angptl3) editing
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
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CN115176001A (zh) 2022-10-11
US20220372483A1 (en) 2022-11-24
TW202136509A (zh) 2021-10-01
BR112022011214A2 (pt) 2022-08-23
AU2020401206A1 (en) 2022-07-14
CO2022009562A2 (es) 2022-08-30
IL293569A (en) 2022-08-01
WO2021119275A1 (en) 2021-06-17
MX2022006950A (es) 2022-11-07
CA3164192A1 (en) 2021-06-17
EP4073249A1 (en) 2022-10-19
KR20220126725A (ko) 2022-09-16
JP2026048746A (ja) 2026-03-17

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