Interrogating Huntington’s in vivo roles through loss of function experiments

Department of Cellular & Physiological Sciences
Seminar Series

Thursday, May 11, 2017
12:30 PM – 1:30 PM
2350 Health Sciences Mall, Vancouver, BC V6T 1Z3

Jeff Carroll, PhD
Associate Professor, Department of Psychology
Behavioral Neuroscience Program, Western Washington University

Expansion of a glutamine-coding CAG trinucleotide near the 5′ end of the Htt gene is the sole cause of Huntington’s Disease – a fatal neurodegenerative disorder. Though the gene was cloned in 1993, the protein remains deeply mysterious – it is extremely large (340kD) and highly conserved, but its precise molecular functions are not clear. We are interrogating huntingtin’s roles using loss of function experiments with antisense oligonucleotides, zinc finger transcriptional repressors and novel tissue-specific knockout mice. We believe our findings are critically important given the rapid advancement of huntingtin lowering therapies in the clinic.