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Researcher Profile: Modifying Disease Progression




Dr. Paolo Moretti is chief of the Sleep & Movement Disorders and Cognitive Neurology Divisions in the Department of Neurology at University of Utah Health and a staff physician at the Salt Lake City Veterans Affairs Medical Center. He received his medical degree in Italy and completed training in neurology, genetics, and research at the University of Michigan, Baylor College of Medicine, and Columbia University. Dr. Moretti sees patients at the University of Utah and the VA and is an investigator in research studies in Parkinson’s disease, Huntington’s disease, essential tremor, and in rare neurogenetic disorders and undiagnosed diseases.

Under his direction, the University of Utah is a study site for the first clinical trial aimed at modifying the progression of Huntington’s Disease (HD). In people with HD, a mistake in the DNA causes the production of a protein called mutant huntingtin (mHTT), which builds up and exerts toxic effects in the brain and spinal cord. Today’s treatments aim to reduce the symptoms of HD, rather than target its cause.

The study currently underway is an investigational therapy based on injection of a short, single-stranded DNA molecule called an antisense oligonucleotide (ASO – see our related feature on this exciting new kind of treatment), designed to target mHTT, reducing its accumulation and toxicity. This study is called GENERATION-HD1 and is a Phase 3 study sponsored by Genentech/Roche.

The study team at the University of Utah comprises the following individuals:

Dr. Paolo Moretti, Principal Investigator
Meghan Zorn, Co-Investigator
Paola Wall, Clinical Research Coordinator
Kendra Fowler, Research Coordinator