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Neil Aronin studies gene silencing to treat Huntington’s disease

There is no treatment and no cure for Huntington’s disease, a fatal genetic disorder that strikes in the prime of one’s life, with symptoms that feel like having ALS, Parkinson’s and Alzheimer’s all at once. Patients suffer a loss of cognition uncontrollable movements and depression, often living out their lives in nursing homes, eventually becoming unable to walk or speak, but often able to comprehend language and recognize family.

Neil Aronin, MD, professor of medicine, co-director of the Neurotherapeutics Institute and a globally recognized leader in the field, is exploring a promising new therapeutic approach to the disease based on gene silencing or RNA interference. The goal of gene silencing is to shut off the mutant huntingtin gene, the one that causes the disease, while preserving a normal huntingtin gene. Initial research indicates that this therapeutic strategy is effective and safe in cultured cells and mice. Dr. Aronin and colleagues are examining how gene silencing can be best delivered to people.

“Scientifically, I’m interested in understanding how we can change or reduce the action of the gene that causes the disease,” Aronin said. “We use RNA interference as the core concept in reducing the human huntingtin. We insert a small piece of RNA into a virus. We can put this virus into the cells in the brain that manifest the disease.”

“Using RNA interference, it reduces the mutant huntingtin expression,” he said. “That’s good news.”

Jason Morgan, 40, has been living with the symptoms of Huntington’s since last year. The disease is hereditary; children born to a parent with the expanded gene have a 50-50 chance of inheriting it. About 30,000 Americans have Huntington’s.

“I woke up one day and all of a sudden, I was feeling suicidal,” Morgan said, describing a common symptom of the disease. “I have never been in that state of mind before. I’m hoping that my son doesn’t have to deal with this. I hope to dear God that he doesn’t have to deal with this.”

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