CN115427570A - 用于靶向pcsk9的组合物和方法 - Google Patents

用于靶向pcsk9的组合物和方法 Download PDF

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Publication number
CN115427570A
CN115427570A CN202180019446.7A CN202180019446A CN115427570A CN 115427570 A CN115427570 A CN 115427570A CN 202180019446 A CN202180019446 A CN 202180019446A CN 115427570 A CN115427570 A CN 115427570A
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sequence
seq
gna
protein
casx
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B·奥克斯
S·希金斯
H·斯平纳
S·丹尼
B·T·斯塔尔
K·泰勒
K·班尼
I·科林
M·阿迪勒
C·乌尔内斯
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Scribe Therapy
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Scribe Therapy
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CN202180019446.7A 2020-01-10 2021-01-08 用于靶向pcsk9的组合物和方法 Pending CN115427570A (zh)

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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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EP (1) EP4087930A1 (https=)
JP (1) JP2023510352A (https=)
KR (1) KR20220125332A (https=)
CN (1) CN115427570A (https=)
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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116978457A (zh) * 2023-09-22 2023-10-31 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及其设计方法
CN118086311A (zh) * 2023-05-25 2024-05-28 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
CN119506351A (zh) * 2024-11-20 2025-02-25 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

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
PH12021553076A1 (en) 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems
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
US20250388885A1 (en) * 2022-03-11 2025-12-25 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
WO2023240027A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Particle delivery systems
WO2023240076A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023240074A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
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
TW202444906A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 用於靶向pcsk9之組合物及方法
WO2024238723A1 (en) * 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
WO2025006782A2 (en) * 2023-06-30 2025-01-02 Chroma Medicine, Inc. Guide rna compositions

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5412087A (en) 1992-04-24 1995-05-02 Affymax Technologies N.V. Spatially-addressable immobilization of oligonucleotides and other biological polymers on surfaces
US5695937A (en) 1995-09-12 1997-12-09 The Johns Hopkins University School Of Medicine Method for serial analysis of gene expression
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
CA2999649A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
CA3038960A1 (en) 2016-09-30 2018-04-05 The Regents Of The University Of California Rna-guided nucleic acid modifying enzymes and methods of use thereof
EP3559223A1 (en) * 2016-12-23 2019-10-30 President and Fellows of Harvard College Gene editing of pcsk9
EP3585894A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders
WO2018195555A1 (en) 2017-04-21 2018-10-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas 9-mediated integration of polynucleotides by sequential homologous recombination of aav donor vectors
US11578334B2 (en) * 2017-10-25 2023-02-14 Monsanto Technology Llc Targeted endonuclease activity of the RNA-guided endonuclease CasX in eukaryotes
WO2019126762A2 (en) * 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12a systems, methods, and compositions for targeted rna base editing
PH12021553076A1 (en) * 2019-06-07 2023-08-14 Scribe Therapeutics Inc Engineered casx systems
CA3153700A1 (en) * 2019-09-09 2021-03-18 Scribe Therapeutics Inc. Compositions and methods for use in immunotherapy

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118086311A (zh) * 2023-05-25 2024-05-28 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
CN116978457A (zh) * 2023-09-22 2023-10-31 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及其设计方法
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
CN119506351A (zh) * 2024-11-20 2025-02-25 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
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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JP2023510352A (ja) 2023-03-13
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AU2021206270A1 (en) 2022-07-21
EP4087930A1 (en) 2022-11-16
WO2021142342A1 (en) 2021-07-15
US20230167424A1 (en) 2023-06-01
IL294620A (en) 2022-09-01

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