DOI
https://doi.org/10.25772/V9PC-A031
Defense Date
2017
Document Type
Thesis
Degree Name
Master of Science
Department
Biochemistry
First Advisor
Gregorio Gil
Abstract
Niemann Pick Type C (NPC) disease is a fatal childhood neurological disease caused by mutations in the NPC-1 protein, resulting in cholesterol buildup in the late endosomes. StarD4 and StarD5 are cholesterol binding proteins that play a role in the intracellular cholesterol transport. In this study we overexpress StarD4 and StarD5 in in vitro and in vivo models, and find evidence of amelioration of the NPC phenotype. This study demonstrates that the overexpression of these proteins has the potential to be a therapeutic treatment for NPC disease.
Rights
© Tavis Sparrer
Is Part Of
VCU University Archives
Is Part Of
VCU Theses and Dissertations
Date of Submission
8-6-2017