Online ISSN: 2515-8260

TARGETING LYSOSOMAL DYSFUNCTION IN WOLMAN DISEASE

Main Article Content

Dr. K. Atchuta Kumar

Abstract

Wolman disease is a rare lysosomal storage disorder caused by mutations in the LIPA gene, resulting in deficient lysosomal acid lipase (LAL) enzyme activity. This deficiency leads to the accumulation of cholesterol esters and triglycerides, causing severe systemic manifestations and a shortened lifespan. This article aims to provide an overview of Wolman disease. Additionally, it explores the potential of pharmacological interventions to target lysosomal dysfunction, reduce lipid accumulation, and ameliorate the clinical manifestations of Wolman disease.

Article Details