Exploiting the Potential of Drosophila Models in Lysosomal Storage Disorders: Pathological Mechanisms and Drug Discovery

Abstract excerpt

Lysosomal storage disorders (LSDs) represent a complex and heterogeneous group of rare genetic diseases due to mutations in genes coding for lysosomal enzymes, membrane proteins or transporters. This leads to the accumulation of undegraded materials within lysosomes and a broad range of severe clinical features, often including the impairment of central nervous system (CNS). When available, enzyme replacement...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Calcium signaling and nucleotide metabolism
• Share to Cellular transport and secretion
• Share to Lysosomal Storage Disorders Research