CN113924368B - 用于抑制scap表达的rnai构建体及其使用方法 - Google Patents

用于抑制scap表达的rnai构建体及其使用方法 Download PDF

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CN113924368B
CN113924368B CN202080041291.2A CN202080041291A CN113924368B CN 113924368 B CN113924368 B CN 113924368B CN 202080041291 A CN202080041291 A CN 202080041291A CN 113924368 B CN113924368 B CN 113924368B
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scap
rnai construct
rnai
expression
nucleotides
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CN113924368A (zh
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A·达斯
B·J·赫贝里奇
J·K·默里
O·霍曼
M·奥尔曼
D·C·H·林
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Amgen Inc
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Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230374522A1 (en) * 2022-04-15 2023-11-23 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating scap activity
US20250313834A1 (en) * 2022-05-25 2025-10-09 Amgen Inc. Rnai constructs for inhibiting scap expression and methods of use thereof
EP4555088A1 (en) * 2022-07-11 2025-05-21 Sanegene Bio USA Inc. Optimized 2'- modified ribose derivatives and methods of use
CN120322552A (zh) * 2022-07-27 2025-07-15 e-生物有限公司 核酸化合物
WO2024104416A1 (zh) * 2022-11-17 2024-05-23 北京福元医药股份有限公司 抑制SCAP基因表达的siRNA、其缀合物和药物组合物及用途

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008036638A2 (en) * 2006-09-18 2008-03-27 Alnylam Pharmaceuticals, Inc. Rnai modulation of scap and therapeutic uses thereof
WO2018039647A1 (en) * 2016-08-26 2018-03-01 Amgen Inc. Rnai constructs for inhibiting asgr1 expression and methods of use thereof

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
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
US5744101A (en) 1989-06-07 1998-04-28 Affymax Technologies N.V. Photolabile nucleoside protecting groups
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
CA2124087C (en) 1991-11-22 2002-10-01 James L. Winkler Combinatorial strategies for polymer synthesis
US5981505A (en) 1993-01-26 1999-11-09 The Trustees Of The University Of Pennsylvania Compositions and methods for delivery of genetic material
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5556752A (en) 1994-10-24 1996-09-17 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US5840710A (en) 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
US5976567A (en) 1995-06-07 1999-11-02 Inex Pharmaceuticals Corp. Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5545531A (en) 1995-06-07 1996-08-13 Affymax Technologies N.V. Methods for making a device for concurrently processing multiple biological chip assays
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US6217900B1 (en) 1997-04-30 2001-04-17 American Home Products Corporation Vesicular complexes and methods of making and using the same
CA2294988C (en) 1997-07-01 2015-11-24 Isis Pharmaceuticals Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
AU747597B2 (en) 1998-07-20 2002-05-16 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US6693187B1 (en) 2000-10-17 2004-02-17 Lievre Cornu Llc Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
CA2487274A1 (en) 2002-05-06 2003-11-13 Nucleonics Inc. Spermine chemically linked to lipids and cell-specific targeting molecules as a transfection agent
US8017762B2 (en) 2003-04-17 2011-09-13 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
CA2685127C (en) 2007-04-23 2019-01-08 Alnylam Pharmaceuticals, Inc. Glycoconjugates of rna interference agents
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
EP3564374A1 (en) 2013-06-21 2019-11-06 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3387129A1 (en) 2015-12-10 2018-10-17 Alnylam Pharmaceuticals, Inc. STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CN110268060B (zh) * 2017-01-10 2024-07-26 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008036638A2 (en) * 2006-09-18 2008-03-27 Alnylam Pharmaceuticals, Inc. Rnai modulation of scap and therapeutic uses thereof
WO2018039647A1 (en) * 2016-08-26 2018-03-01 Amgen Inc. Rnai constructs for inhibiting asgr1 expression and methods of use thereof

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IL288398A (en) 2022-01-01
US20220307022A1 (en) 2022-09-29
CA3141902A1 (en) 2020-12-03
TW202111124A (zh) 2021-03-16
AU2020284254A1 (en) 2021-12-23
CL2021003169A1 (es) 2023-01-20
AR119061A1 (es) 2021-11-17
UY38733A (es) 2020-11-30
SG11202113112WA (en) 2021-12-30
JP2026016500A (ja) 2026-02-03
CN113924368A (zh) 2022-01-11
EA202193296A1 (ru) 2022-03-10
WO2020243702A2 (en) 2020-12-03
WO2020243702A3 (en) 2021-04-22
CN119842815A (zh) 2025-04-18
MX2021014465A (es) 2022-01-06
KR20220016138A (ko) 2022-02-08
EP3976786A2 (en) 2022-04-06
MX2026000159A (es) 2026-03-02
JP7763104B2 (ja) 2025-10-31
CL2025000194A1 (es) 2025-06-23
BR112021024080A2 (pt) 2022-02-08

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