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

Included in

Biochemistry Commons

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