Graduate Students

	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, FL 

Research interests

RNAi, translational repression, genome editing, gene therapy  

Project

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.