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Recent advances in novel therapies for lipid disorders

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Article (526.9Kb)
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final draft
Date
2019
Author(s)
Tirronen, Annakaisa
Hokkanen, Krista
Vuorio, Taina
Ylä-Herttuala, Seppo
Unique identifier
10.1093/hmg/ddz132
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Citation
Tirronen, Annakaisa. Hokkanen, Krista. Vuorio, Taina. Ylä-Herttuala, Seppo. (2019). Recent advances in novel therapies for lipid disorders.  Human molecular genetics, 28 (R1) , R49-R54. 10.1093/hmg/ddz132.
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Abstract

The prevalence of lipid disorders is alarmingly increasing in the Western world. They are the result of either primary causes, such as unhealthy lifestyle choices or inherited risk factors, or secondary causes like other diseases or medication. Atypical changes in the synthesis, processing and catabolism of lipoprotein particles may lead to severe hypercholesterolemia, hypertriglyceridemia or elevated Lp(a). Although cholesterol-lowering drugs are the most prescribed medications, not all patients achieve guideline recommended cholesterol levels with the current treatment options, emphasising the need for new therapies. Also, some lipid disorders do not have any treatment options but rely only on stringent dietary restriction. Patients with untreated lipid disorders carry a severe risk of cardiovascular disease, diabetes, non-alcoholic fatty liver disease and pancreatitis among others. To achieve better treatment outcome, novel selective gene expression and epigenetic targeting therapies are constantly being developed. Therapeutic innovations employing targeted RNA technology utilise small interfering RNAs, antisense oligonucleotides, long non-coding RNAs and microRNAs to regulate target protein production whereas viral gene therapy provides functional therapeutic genes and CRISPR/Cas technology relies on gene editing and transcriptional regulation. In this review, we will discuss the latest advances in clinical trials for novel lipid-lowering therapies and potential new targets in pre-clinical phase.

Subjects
pancreatitis   hypercholesterolemia   lipoproteins   hypertriglyceridemia   cardiovascular disease risk factors   gene therapy   gene expression   antisense oligonucleotides   diabetes mellitus   type 2   diet   anticholesteremic agents   genes   viral   life style   western world   cholesterol measurement test   treatment outcome   lipid-lowering therapy   lipid disorders   rna   non-alcoholic fatty liver disease   transcriptional control   epigenetics   micrornas   catabolism   crispr   gene editing   
URI
https://erepo.uef.fi/handle/123456789/7808
Link to the original item
http://dx.doi.org/10.1093/hmg/ddz132
Publisher
Oxford University Press (OUP)
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  • Terveystieteiden tiedekunta [1324]
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