A Journey towards Understanding the Molecular Pathology and Developing Therapies for Lysosomal Storage Disorders

Cells. 2021 Dec 23;11(1):36. doi: 10.3390/cells11010036.

Abstract

Lysosomal storage disorders (LSDs) are rare, monogenic diseases characterized by aberrant lysosomes with storage material [...].

Publication types

  • Editorial
  • Introductory Journal Article

MeSH terms

  • Animals
  • Autophagy
  • Clinical Trials as Topic
  • Disease Models, Animal
  • Enzyme Replacement Therapy
  • Humans
  • Lysosomal Storage Diseases / therapy*
  • Organelles / metabolism
  • Pathology, Molecular*