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

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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Detalles Bibliográficos
Autores principales: Zancanella, Vanessa, Vallès, Astrid, Liefhebber, Jolanda M.P., Paerels, Lieke, Tornero, Carlos Vendrell, Wattimury, Hendrina, van der Zon, Tom, van Rooijen, Kristel, Golinska, Monika, Grevelink, Tamar, Ehlert, Erich, Pieterman, Elsbet Jantine, Keijzer, Nanda, Princen, Hans Marinus Gerardus, Stokman, Geurt, Liu, Ying Poi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10165407/
https://www.ncbi.nlm.nih.gov/pubmed/37168797
http://dx.doi.org/10.1016/j.omtn.2023.04.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10165407/
https://www.ncbi.nlm.nih.gov/pubmed/37168797
http://dx.doi.org/10.1016/j.omtn.2023.04.004

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