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Article

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

Current opinion in lipidology - 1 Dec 2021

McIntosh Craig S, Watts Gerald F, Wilton Steve D, Aung-Htut May T

Abstract excerpt

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 hypercholesterolemic patients carrying mutations that compromise normal splicing of the low-density lipoprotein receptor (LDLR)...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 34653074
DOI: 10.1097/MOL.0000000000000793
PMCID: PMC8631153

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