Date of Award

2024

Degree Type

Thesis

Degree Name

Master of Science in Biological and Environmental Sciences (MSBES)

Specialization

Cell and Molecular Biology

Department

Cell & Molecular Biology

First Advisor

Niall G. Howlett

Abstract

Fanconi anemia (FA) is a rare human genetic disease characterized by bone marrow failure, congenital abnormalities, predisposition to cancer, accelerated aging, and premature mortality. FA is caused by mutations in any one of 23 genes. The FA proteins play a crucial role in the repair of DNA interstrand cross-links (ICLs) and the maintenance of genome stability. Recently, central nervous system (CNS) defects have become increasingly observed among FA patients. These include acute or chronic loss of neurological capabilities, accumulation of white matter lesions and large pseudo-tumor lesions with vasogenic edema, and retinal vasculopathy. These neurological manifestations have been coined Fanconi Anemia Neurological Syndrome (FANS). The molecular origins of FANS are unknown. Recent data from our lab and others shows that, under conditions of replication stress, the FANCD2 protein binds to several transcriptionally active large neural genes. Many of these genes encode for proteins that function in neuronal differentiation, migration, and cell adhesion, and many are linked to neurodevelopmental and neuropsychiatric disorders. We hypothesize that FANCD2 may play an important role in the maintenance of genome stability during neurogenesis, when neural stem and progenitor cells are undergoing rapid proliferation and expansion. To study the role of FANCD2 in neuronal differentiation, we have developed a lentiviral doxycycline-inducible shRNA system to inducibly deplete FANCD2 in the SH-SY5Y neuroblastoma line. This system will allow us to determine if loss of the FANCD2 protein adversely impacts the ability of SH-SY5Y cells, as well as other cells, to differentiate into mature neurons.

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