KR102907245B1 - 혈색소병증 치료를 위한 시스템 및 방법 - Google Patents

혈색소병증 치료를 위한 시스템 및 방법

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Publication number
KR102907245B1
KR102907245B1 KR1020207029274A KR20207029274A KR102907245B1 KR 102907245 B1 KR102907245 B1 KR 102907245B1 KR 1020207029274 A KR1020207029274 A KR 1020207029274A KR 20207029274 A KR20207029274 A KR 20207029274A KR 102907245 B1 KR102907245 B1 KR 102907245B1
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certain embodiments
grna
rna
target region
cpf1
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KR20200132924A (ko
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제니퍼 리 고리
에두아르드 오페핀 데 라모드-드뤼지
잭 헤스
존 앤서니 주리스
카이신 창
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에디타스 메디신, 인코포레이티드
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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KR1020207029274A 2018-03-14 2019-03-14 혈색소병증 치료를 위한 시스템 및 방법 Active KR102907245B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020257043580A KR20260006713A (ko) 2018-03-14 2019-03-14 혈색소병증 치료를 위한 시스템 및 방법

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862643168P 2018-03-14 2018-03-14
US62/643,168 2018-03-14
US201862767488P 2018-11-14 2018-11-14
US62/767,488 2018-11-14
US201862773073P 2018-11-29 2018-11-29
US62/773,073 2018-11-29
PCT/US2019/022374 WO2019178426A1 (en) 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies

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KR20200132924A KR20200132924A (ko) 2020-11-25
KR102907245B1 true KR102907245B1 (ko) 2026-01-05

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KR1020257043580A Pending KR20260006713A (ko) 2018-03-14 2019-03-14 혈색소병증 치료를 위한 시스템 및 방법

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US (2) US12031132B2 (enExample)
EP (1) EP3765614A1 (enExample)
JP (2) JP7706888B2 (enExample)
KR (2) KR102907245B1 (enExample)
CN (1) CN112020558A (enExample)
AU (2) AU2019236209B2 (enExample)
BR (1) BR112020018364A2 (enExample)
CA (1) CA3093702A1 (enExample)
IL (2) IL277234B2 (enExample)
MX (1) MX2020009487A (enExample)
SG (1) SG11202008956XA (enExample)
WO (1) WO2019178426A1 (enExample)

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US20230279438A1 (en) * 2020-05-13 2023-09-07 Inserm (Institut National De La Santé Et La Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
JP7753209B2 (ja) * 2020-11-18 2025-10-14 エディタス・メディシン、インコーポレイテッド 異常ヘモグロビン症の治療のための改変細胞及び方法
JP2024506645A (ja) * 2021-02-11 2024-02-14 コーニンクレッカ ネザーランド アカデミー ヴァン ウェテンシャッペン 転写調節遺伝子の編集による疾患の治癒
EP4047095A1 (en) * 2021-02-22 2022-08-24 Eberhard Karls Universität Tübingen Medizinische Fakultät Method and composition for a targeted gene knockout
JP2024528967A (ja) * 2021-08-02 2024-08-01 エディタス・メディシン、インコーポレイテッド ヘモグロビン異常症の治療のためのシステム及び方法
CN115851706A (zh) * 2021-09-24 2023-03-28 华东师范大学 一种碱基编辑系统及其应用
KR102853682B1 (ko) 2022-03-07 2025-09-03 광주과학기술원 신속 DNA 진단을 위한 Cpf1 변이 단백질
CN121241136A (zh) * 2023-04-07 2025-12-30 基因泰克公司 经修饰的指导rna
EP4712948A2 (en) * 2023-05-17 2026-03-25 Renagade Therapeutics Management Inc. Gene editing systems and compositions for treatment of hemoglobinopathies and methods of using the same
WO2025067403A1 (zh) * 2023-09-28 2025-04-03 上海天泽云泰生物医药有限公司 用于治疗血红蛋白疾病的组合物和方法
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
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

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