JP2023510352A5 - - Google Patents

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Publication number
JP2023510352A5
JP2023510352A5 JP2022542340A JP2022542340A JP2023510352A5 JP 2023510352 A5 JP2023510352 A5 JP 2023510352A5 JP 2022542340 A JP2022542340 A JP 2022542340A JP 2022542340 A JP2022542340 A JP 2022542340A JP 2023510352 A5 JP2023510352 A5 JP 2023510352A5
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JP
Japan
Prior art keywords
sequence
improved
protein
seq
nucleic acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022542340A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023510352A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/012804 external-priority patent/WO2021142342A1/en
Publication of JP2023510352A publication Critical patent/JP2023510352A/ja
Publication of JP2023510352A5 publication Critical patent/JP2023510352A5/ja
Pending legal-status Critical Current

Links

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)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (2)

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

Family

ID=74505368

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022542340A Pending JP2023510352A (ja) 2020-01-10 2021-01-08 Pcsk9の標的化のための組成物および方法

Country Status (9)

Country Link
US (1) US20230167424A1 (enExample)
EP (1) EP4087930A1 (enExample)
JP (1) JP2023510352A (enExample)
KR (1) KR20220125332A (enExample)
CN (1) CN115427570A (enExample)
AU (1) AU2021206270A1 (enExample)
CA (1) CA3163714A1 (enExample)
IL (1) IL294620A (enExample)
WO (1) WO2021142342A1 (enExample)

Families Citing this family (19)

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US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
EP3980533A1 (en) 2019-06-07 2022-04-13 Scribe Therapeutics Inc. Engineered casx systems
KR20230128289A (ko) 2020-12-03 2023-09-04 스크라이브 테라퓨틱스 인크. 조작된 클래스 2 유형 v crispr 시스템
WO2022261149A2 (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
WO2023235818A2 (en) * 2022-06-02 2023-12-07 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
WO2023235888A2 (en) 2022-06-03 2023-12-07 Scribe Therapeutics Inc. COMPOSITIONS AND METHODS FOR CpG DEPLETION
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
TW202413643A (zh) 2022-06-07 2024-04-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
US20250361525A1 (en) 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
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
PE20252746A1 (es) 2023-03-29 2025-12-05 Scribe Therapeutics Inc Composiciones y metodos para el direccionamiento a 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
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, 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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US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
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WO2010075303A1 (en) 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
BR112018008971A2 (pt) * 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
MX2019003674A (es) 2016-09-30 2021-01-08 Univ California Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas.
KR20230125856A (ko) * 2016-12-23 2023-08-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Pcsk9의 유전자 편집
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US20210079366A1 (en) * 2017-12-22 2021-03-18 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
EP3980533A1 (en) * 2019-06-07 2022-04-13 Scribe Therapeutics Inc. Engineered casx systems
WO2021050601A1 (en) * 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy

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