Graduate Students
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Gabriela Toro
Graduate Student since 2012
"I find myself extremely fortunate. Being part of both a neurology and gene therapy lab, has given me the opportunity to translate my findings into a possible therapy for ALS patients. Seeing the progression of the disease and the lack of a cure or treatment is a strong motivation for my research."
Education
2010 BSc in Biology, Barry University, Miami Shores, FLResearch interests
RNAi, translational repression, genome editing, gene therapyProject
Gene therapy for amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects upper and lower motor neurons. The project is specifically focused on a hexanucleotide repeat mutation found in chromosome 9 open reading frame 72 (C9orf72). Although the underlying cause for disease remains unknown, existing hypothesis of haploinsufficiency, RNA gain of function and RAN translation have been widely explored. We use recombinant adeno-associated virus (rAAV) as vehicles to deliver engineered microRNAs against C9orf72 toxic variants both in vitro in cell lines as well as in vivo in a C9orf72 transgenic mouse model.
