JP2023510352A - Ｐｃｓｋ９の標的化のための組成物および方法 - Google Patents

Ｐｃｓｋ９の標的化のための組成物および方法 Download PDF

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JP2023510352A
JP2023510352A JP2022542340A JP2022542340A JP2023510352A JP 2023510352 A JP2023510352 A JP 2023510352A JP 2022542340 A JP2022542340 A JP 2022542340A JP 2022542340 A JP2022542340 A JP 2022542340A JP 2023510352 A JP2023510352 A JP 2023510352A
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gna
sequence
seq
protein
casx
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JP2023510352A5 (enExample
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オークス，ベンジャミン
ヒギンズ，ショーン
スピナー，ハンナ
デニー，サラ
ティ スタール，ブレット
テイラー，キアン
バニー，キャサリン
コリン，イザベル
アディル，マルーフ
アーンズ，コール
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Scribe Therapeutics Inc
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Scribe Therapeutics Inc
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JP2022542340A 2020-01-10 2021-01-08 Ｐｃｓｋ９の標的化のための組成物および方法 Pending JP2023510352A (ja)

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US202062959685P 2020-01-10 2020-01-10
US62/959,685 2020-01-10
PCT/US2021/012804 WO2021142342A1 (en) 2020-01-10 2021-01-08 Compositions and methods for the targeting of pcsk9

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JP2023510352A true JP2023510352A (ja) 2023-03-13
JP2023510352A5 JP2023510352A5 (enExample) 2024-01-16

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US (1) US20230167424A1 (enExample)
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KR (1) KR20220125332A (enExample)
CN (1) CN115427570A (enExample)
AU (1) AU2021206270A1 (enExample)
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AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2022261150A2 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Particle delivery systems
WO2023173110A1 (en) * 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
CA3257824A1 (en) * 2022-06-02 2023-12-07 Scribe Therapeutics Inc. MODIFIED TYPE V, CLASS 2 CRISPR SYSTEMS
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
EP4536286A1 (en) 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
JP2025532127A (ja) 2022-09-23 2025-09-29 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｍｅｃｐ２発現を低減する化合物及び方法
WO2024206555A1 (en) * 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2024238723A1 (en) * 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
CN118086311B (zh) * 2023-05-25 2024-08-09 苏州时安生物技术有限公司 抑制PCSK9基因表达的siRNA、其缀合物和药物组合物及用途
EP4735603A2 (en) * 2023-06-30 2026-05-06 nChroma Bio, Inc. Guide rna compositions
CN116978457B (zh) * 2023-09-22 2023-12-22 成都斯马特科技有限公司 避免rna检测过程中假基因干扰的引物和探针组合及其设计方法
CN119506351B (zh) * 2024-11-20 2025-08-19 中国人民解放军陆军军医大学第二附属医院 一体化pcsk9腺嘌呤碱基编辑系统及应用

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