Related Experiment Video
Updated: Mar 8, 2026

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
Published on: July 23, 2016
Lysosomal Proteins as a Therapeutic Target in Neurodegeneration
Jessica M Mc Donald1, Dimitri Krainc1
1Department of Neurology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois 60611-4296; email: jessica.mcdonald@northwestern.edu , krainc@northwestern.edu.
Abstract:
Several proteins that are mutated in lysosomal storage diseases are linked to neurodegenerative disease. This review focuses on some of these lysosomal enzymes and transporters, as well as current therapies that have emerged from the lysosomal storage disease field. Given the deeper genetic understanding of lysosomal defects in neurodegeneration, we explore why some of these orphan disease drug candidates are also attractive targets in subpopulations of individuals with neurodegenerative disease.
Related Concept Videos
Lysosomal Hydrolases
The Proteasome
In this pathway, the target proteins are first tagged with small proteins called ubiquitin. This involves participation of a series of enzymes including— E1 (ubiquitin-activating enzyme), E2 (ubiquitin-conjugating enzyme), and E3...
The Proteasome
In this pathway, the target proteins are first tagged with small proteins called ubiquitin. A series of enzymes carry out the ubiquitination of the target proteins - E1 (ubiquitin-activating enzyme), E2 (ubiquitin-conjugating enzyme), and E3...
Parkinson's Disease: Overview

