Key result
Canonical pathway mutations drive monogenic and polygenic hypertriglyceridemia, supporting genetic screening and targeted therapies.
Why the study?
Hypertriglyceridemia is a complex metabolic disorder with heterogeneous phenotypic expression, warranting an integrated review of its genetic basis and recent progress in triglyceride-lowering treatments.
Understanding the genetic underpinnings of both monogenic and polygenic hypertriglyceridemia provides critical pathophysiologic insights that open new translational opportunities for genetic screening and novel targeted lipid-lowering therapies.
Answers are grounded in this paper and cite their sources.
Hypothesis-generating for genetic screening in hypertriglyceridemia; leaves open prospective validation of targeted therapies.
Hypertriglyceridemia is an exceptionally complex metabolic disorder characterized by elevated plasma triglycerides associated with an increased risk of acute pancreatitis and cardiovascular diseases such as coronary artery disease. Its phenotype expression is widely heterogeneous and heavily influenced by conditions as obesity, alcohol consumption, or metabolic syndromes. Looking into the genetic underpinnings of hypertriglyceridemia, this review focuses on the genetic variants in LPL, APOA5, APOC2, GPIHBP1 and LMF1 triglyceride-regulating genes reportedly associated with abnormal genetic transcription and the translation of proteins participating in triglyceride-rich lipoprotein metabolism. Hypertriglyceridemia resulting from such genetic abnormalities can be categorized as monogenic or polygenic. Monogenic hypertriglyceridemia, also known as familial chylomicronemia syndrome, is caused by homozygous or compound heterozygous pathogenic variants in the five canonical genes. Polygenic hypertriglyceridemia, also known as multifactorial chylomicronemia syndrome in extreme cases of hypertriglyceridemia, is caused by heterozygous pathogenic genetic variants with variable penetrance affecting the canonical genes, and a set of common non-pathogenic genetic variants (polymorphisms, using the former nomenclature) with well-established association with elevated triglyceride levels. We further address recent progress in triglyceride-lowering treatments. Understanding the genetic basis of hypertriglyceridemia opens new translational opportunities in the scope of genetic screening and the development of novel therapies.
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Alves et al. (2024) conducted a review in Hypertriglyceridemia. Genetic variants in LPL, APOA5, APOC2, GPIHBP1, and LMF1 was evaluated. Genetic abnormalities in LPL, APOA5, APOC2, GPIHBP1, and LMF1 underlie both monogenic and polygenic forms of hypertriglyceridemia, driving the clinical phenotype and opening opportunities for genetic screening and targeted therapies.
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