Scientists at the Gladstone Institutes and an international team of researchers have generated a human model of Huntington’s disease—directly from the skin cells of patients with the disease.
For years, scientists have studied Huntington’s disease primarily in post-mortem brain tissue or laboratory animals modified to mimic the disease. Today, in Cell Stem Cell, the international team shows how they developed a human model of Huntington’s disease, which causes a diverse range of neurological impairments. The new model should help scientists better understand the development of Huntington’s—and provide better ways to identify and screen potential therapeutics for this devastating disease.
“An advantage of this human model is that we now have the ability to identify changes in brain cells over time—during the degeneration process and at specific stages of brain-cell development,” said Gladstone Senior Investigator Steve Finkbeiner, MD, PhD. “We hope this model will help us more readily uncover relevant factors that contribute to Huntington’s disease and especially to find successful therapeutic approaches.”