Gene-based therapies in lipidology: current status and future challenges

Current Opinion in Lipidology
Daniel Gaudet, Diane Brisson

Abstract

Gene-based therapies are designed to modulate gene expression in specific tissues by introducing into cells transgenes, antisense oligonucleotides, RNA interference, microRNAs, or a variety of other oligonucleotide-based compounds and their delivery systems. Several types of gene-based therapies are already available or in clinical development to treat severe lipid-related disorders and associated risk. The review briefly presents the current status and future challenges of these therapies in clinical lipidology, focusing on most advanced and promising agents or mechanisms. Gene-based agents address several unmet medical needs in lipidology such as homozygous familial hypercholesterolemia, familial or multifactorial chylomicronemia, severe hypertriglyceridemia, elevated lipoprotein (a), familial partial lipodystrophy, nonalcoholic fatty liver disease, and hypoalphalipoproteinemia. Most advanced antisense oligonucleotide drugs target apolipoprotein C-III, apolipoprotein (a), angiopoietin-like 3, and diacylglycerol o-acyltransferase-2. Long-term efficacy and safety data are now available for two gene-based agents, mipomersen, approved in the USA for homozygous familial hypercholesterolemia and Glybera, AAV1-LPLS447X gene therapy,...Continue Reading

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Aug 20, 2016·Human Molecular Genetics·Lon R Cardon, Tim Harris
Mar 3, 2017·The Tohoku Journal of Experimental Medicine·Cui-Zhong LiuYu-Qing Huang
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Methods Mentioned

BETA
antisense oligonucleotides
FCS
exome sequencing
gene replacement therapy

Software Mentioned

GalNac

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