Graduation Year
2020
Document Type
Master's Thesis
Degree
Master of Science
Program
Biological Science
Program Director
Meredith Protas, PhD
First Reader
Manuel Lopez, PhD
Second Reader
Kenneth Frost, PhD
Abstract
Neurofilament light chain (NF-L), a protein that is found in the axon cytoskeleton of neurons, can become elevated in both cerebrospinal fluid (CSF) and blood in response to brain injury or neurodegenerative disease progression. Thus, NF-L can be a useful fluid biomarker for tracking the progression and correction of a neurodegenerative disorder. However, NF-L response to disease modification and treatments has not yet been fully characterized. This thesis offers new insight into the behavior of NF-L in mouse models of severe progressive central nervous system neurodegeneration. Herein, we aimed to characterize the levels of NF-L during lysosomal storage disease progression and the response of NF-L with enzyme replacement treatment.