TW202028460A - 用於乳酸脫氫酶(ldha)基因編輯之組合物及方法 - Google Patents

用於乳酸脫氫酶(ldha)基因編輯之組合物及方法 Download PDF

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TW202028460A
TW202028460A TW108135340A TW108135340A TW202028460A TW 202028460 A TW202028460 A TW 202028460A TW 108135340 A TW108135340 A TW 108135340A TW 108135340 A TW108135340 A TW 108135340A TW 202028460 A TW202028460 A TW 202028460A
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seq
composition
guide
sequence
sequence selected
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柴克瑞 威廉 岱美克
書布 歐德
安妮特 胡伯納
斯里加尼 斯里哈爾
布萊德利 安德魯 莫雷
瓦特 史瑞普斯
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美商英特利亞醫療公司
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TW108135340A 2018-09-28 2019-09-27 用於乳酸脫氫酶(ldha)基因編輯之組合物及方法 TW202028460A (zh)

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US201862738956P 2018-09-28 2018-09-28
US62/738,956 2018-09-28
US201962834334P 2019-04-15 2019-04-15
US62/834,334 2019-04-15
US201962841740P 2019-05-01 2019-05-01
US62/841,740 2019-05-01

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US20230034581A1 (en) 2021-06-04 2023-02-02 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof
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WO2023122433A1 (en) * 2021-12-22 2023-06-29 Arbor Biotechnologies, Inc. Gene editing systems targeting hydroxyacid oxidase 1 (hao1) and lactate dehydrogenase a (ldha)
US20250032642A1 (en) 2022-02-02 2025-01-30 Regeneron Pharmaceuticals, Inc. Crispr-mediated transgene insertion in neonatal cells
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JP2025514304A (ja) 2022-04-29 2025-05-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 遺伝子治療法のための組織特異的遺伝子外セーフハーバーの同定
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TW202519551A (zh) 2023-07-28 2025-05-16 美商雷傑納榮製藥公司 用於治療酸性神經髓磷脂酶缺乏症之抗ＴｆＲ：酸性神經髓磷脂酶
TW202513801A (zh) 2023-07-28 2025-04-01 美商雷傑納榮製藥公司 用於治療龐貝氏症之抗ＴｆＲ：ＧＡＡ及抗ＣＤ６３：ＧＡＡ插入
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WO2025162435A1 (en) * 2024-02-02 2025-08-07 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of liver disease
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AU2019347517A1 (en) 2021-05-06
KR20210086621A (ko) 2021-07-08
BR112021005718A2 (pt) 2021-06-22
CA3114425A1 (en) 2020-04-02
EP3856901A1 (en) 2021-08-04
PH12021550686A1 (en) 2022-02-28
JP2022502055A (ja) 2022-01-11
SG11202102660RA (en) 2021-04-29
CO2021005231A2 (es) 2021-06-10
IL281529A (en) 2021-05-31
US20210222173A1 (en) 2021-07-22
CN113056559A (zh) 2021-06-29
WO2020069296A1 (en) 2020-04-02

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