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Splice correction therapies for familial hypercholesterolemic patients with low-density lipoprotein receptor mutations

PURPOSE OF REVIEW: Antisense oligomers (ASOs) have been available for decades: however, only recently have these molecules been applied clinically. This review aims to discuss the possible development of antisense-mediated splice correction therapies as precision medicines for familial hypercholeste...

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Detalles Bibliográficos
Autores principales:	McIntosh, Craig S., Watts, Gerald F., Wilton, Steve D., Aung-Htut, May T.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Lippincott Williams & Wilkins 2021
Materias:	THERAPY AND CLINICAL TRIALS: Edited by Erik S.G. Stroes and Gerald F. Watts
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8631153/ https://www.ncbi.nlm.nih.gov/pubmed/34653074 http://dx.doi.org/10.1097/MOL.0000000000000793

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