UC Davis’ leadership in Huntington’s disease research received another boost recently when Kyle Fink, a post-doctoral fellow at the Institute for Regenerative Cures, was recognized by the American Academy of Neurology (AAN) for the cutting-edge research findings in gene editing that he presented at the 2015 Society for Neuroscience Annual Meeting.
The academy’s newsletter, Neurology Today, featured Fink and his colleagues for their gene-editing technique that resulted in a significant reduction in the aggregated mutant huntingtin protein, which has been implicated in the devastating neurodegenerative disorder. Their research is one of the offshoots of a multi–million state stem cell grant to UC Davis that is leading up to what soon is expected to be the world’s first FDA-approved cellular therapy for Huntington’s disease patients.
“It’s thrilling to see Kyle’s work selected from among thousands of submissions to this prestigious international neuroscience meeting,” said Vicki Wheelock, health sciences clinical professor of neurology and director of the Huntington’s Disease Society of America Center of Excellence at UC Davis.
“The laboratory work Kyle and his team are performing under the mentorship of Dr. David Segal and Dr. Jan Nolta is an exciting new approach to attacking Huntington’s disease at the level of the disease-causing mutation,” added Wheelock. “Once the technique is refined, the team will investigate the best way to deliver gene-editing therapy to its target in the brain.”
Fink, who works at Nolta’ s laboratory in Sacramento, is investigating the use of transcription-like effectors (TALEs) in human Huntington’s disease fibroblasts and neurons to see if they can reduce the production of the mutant huntingtin protein that leads to brain degeneration and eventual death in patients with HD. TALEs can be designed to target single nucleotide polymorphisms in the mutant allele, causing either a collapse in the expanded mutant allele or repressing the mutant gene. One advantage of their approach is that it can selectively target the affected allele and not the normal copy of the gene.
“Kyle’s gene-editing success provides a foundational pathway for the human clinical trials we hope to launch in the next few years,” said Wheelock, who is co-principal investigator on the stem cell grant with Nolta.