Proof-of-concept study for liver-directed miQURE technology in a dyslipidemic mouse model

Proof-of-concept study for liver-directed miQURE technology in a dyslipidemic mouse model

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A gene-silencing platform (miQURE) has been developed and successfully used to deliver therapeutic microRNA (miRNA) to the brain, reducing levels of neurodegenerative disease-causing proteins/RNAs via RNA interference and improving the disease phenotype in animal models. This study evaluates the use...

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Bibliographic Details
Main Authors: Vanessa Zancanella, Astrid Vallès, Jolanda M.P. Liefhebber, Lieke Paerels, Carlos Vendrell Tornero, Hendrina Wattimury, Tom van der Zon, Kristel van Rooijen, Monika Golinska, Tamar Grevelink, Erich Ehlert, Elsbet Jantine Pieterman, Nanda Keijzer, Hans Marinus Gerardus Princen, Geurt Stokman, Ying Poi Liu
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Non-coding RNAs
gene therapy
gene-silencing
miRNA
RNA interference
AAV
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123000902

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http://www.sciencedirect.com/science/article/pii/S2162253123000902

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