Regulatory Non-Coding RNAs in Familial Hypercholesterolemia, Theranostic Applications

Area of Science:

• Biochemistry
• Genetics
• Molecular Biology

Background:

• Familial hypercholesterolemia (FH) is a monogenic disorder characterized by elevated low-density lipoprotein cholesterol (LDL-C) levels.
• FH significantly increases the risk of atherosclerosis and cardiovascular disease (CVD), necessitating early diagnosis and intervention.
• Non-coding RNAs (ncRNAs) are increasingly recognized for their roles in regulating cellular processes and as potential biomarkers.

Purpose of the Study:

• To review the pathophysiology of FH and current therapeutic strategies.
• To explore the emerging role of ncRNAs in FH diagnosis and treatment.
• To compare the advantages and disadvantages of ncRNA-based therapies with conventional treatments for FH.

Main Methods:

• Literature review of FH mechanisms, pathophysiology, and therapeutic approaches.
• Analysis of studies investigating ncRNAs in LDL-C metabolism and FH.
• Comparative discussion of ncRNA-based theranostics versus established therapies.

Main Results:

• FH is driven by genetic defects affecting LDL-C metabolism, leading to premature cardiovascular events.
• ncRNAs play crucial roles in maintaining LDL-C homeostasis and are implicated in FH pathogenesis.
• ncRNAs show promise as diagnostic biomarkers and therapeutic targets for FH.

Conclusions:

• Targeting ncRNAs presents a novel theranostic avenue for managing FH and its associated cardiovascular risks.
• ncRNA-based strategies offer potential advantages over conventional treatments but require further investigation.
• Early identification and intervention using ncRNA biomarkers and therapeutics can improve FH patient outcomes.