Cholesterol Regulation in Familial Hypercholesterolemia by Long Noncoding RNA

Abstract

Familial hypercholesterolemia is one of the FY21 PRMRP Topic Areas. Patients with familial hypercholesterolemia have a very high level of cholesterol in their blood, which may lead to a life-threatening condition. Reducing blood cholesterol levels is the primary goal to treat familial hypercholesterolemia, which can be achieved by inhibiting cholesterol synthesis in the liver. Statins that inhibit cholesterol synthesis in the liver can reduce blood cholesterol levels. Thus, statins have been used to treat familial hypercholesterolemia. However, some patients have no or low response to statin therapy. Therefore, there is an urgent need to develop new therapeutic approaches. Long non-coding RNAs are a novel class of genes that have attracted a lot of attention in the development of novel therapeutics for human disease. However, the therapeutic roles of long non-coding RNAs in familial hypercholesterolemia are largely unknown. In our preliminary study, we identified that a long non-coding RNA plays a critical role in liver cholesterol synthesis. We hypothesize that long non-coding RNA that inhibits cholesterol synthesis in the liver can be used to develop novel therapeutics for familial hypercholesterolemia. In this proposal, we will use a series of molecular, cell biological, and familial hypercholesterolemia mouse models to test our hypothesis. Completion of this study may open new revenues to develop therapeutic intervention for familial hypercholesterolemia by using long non-coding RNA.

Document Details

Document Type
DoD Grant Award
Publication Date
Dec 28, 2022
Source ID
W81XWH2210088

Entities

People

  • Ling Yang

Organizations

  • Temple University
  • United States Army

Tags

Readers

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  • Oncology
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