Professor, UF Scripps, Florida, USA
CEO, Vova Ida Therapeutics, Inc. (VIT)
Research Focus: Neurodegenerative diseases
Prion diseases are fatal neurodegenerative diseases affecting humans and animals. There is no cure for prion diseases. They are caused by an unconventional infectious agent called prion, which is thought to be composed mainly by a misfolded form of a host protein, the prion protein (PrP). In humans, prions cause Creutzfeldt-Jakob disease; in animals, scrapie has been known to ravage sheep flocks for two hundred years, and chronic wasting disease is of concern in the United States. The recent epidemic of bovine spongiform encephalopathy in the United Kingdom has caused major turmoil throughout Europe, and later, in other countries such as Japan, Canada and the United States, because the bovine prion disease is transmissible to humans causing variant Creutzfeldt-Jakob disease. The transmissibility of the latter by blood transfusion created a novel public health issue.
Recent years have witnessed major advances in our understanding of prion disease mechanisms. Prion diseases belong to the same group of protein misfolding neurodegenerative diseases as Alzheimer’s, Parkinson’s, Huntington’s diseases, amyotrophic lateral sclerosis (ALS) and others. In all these diseases, a host protein misfolds, aggregates and becomes toxic to neurons. Protein clumps propagate from cell to cell in a “prion-like” manner. In our research program, we investigate which form(s) of these culprit proteins cause the death of neurons, and by which mechanisms. We also run a drug discovery and development program focusing on new intervention strategies to slow or stop disease progression and support neuronal regeneration.