HudsonAlpha Institute for Biotechnology and CHDI Foundation have announced a research collaboration to study the gene regulatory network controlling the expression of the huntingtin (HTT) gene that, when mutated, causes Huntington disease, a neurodegenerative disorder characterized by the progressive breakdown of nerve cells in the brain. Richard Myers, PhD, president of HudsonAlpha is leading the project along with senior scientist Brian Roberts.
"While we know that Huntington disease is caused by a mutation in a single inherited gene, we want to learn more about the molecular network that controls the way instructions in the HTT gene affect specific cell functions," Myers said.
More than 30,000 Americans suffer from Huntington disease, and another 200,000 are at risk for inheriting the disease. Every child who has a parent with Huntington disease has a 50/50 chance of also inheriting the disease gene, and everyone who inherits the disease gene develops the fatal disorder. While early symptoms include personality changes, forgetfulness, unsteady gait and slurred speech, over time, the disease affects the ability to reason, walk and speak. Ultimately, patients succumb to pneumonia, heart failure or other complications.
"A thorough understanding of the molecular network that regulates the HTT gene could eventually lead to therapeutic interventions for individuals suffering from the disorder," Myers said.