CHOP gene therapy expert fine-tunes protein signals, improves motor function and reduces brain shrinkage in a neurological disorder IMAGE:Beverly L. Davidson, Ph.D., a gene therapy expert, is the director of The Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia. view more Credit: The Children's Hospital of Philadelphia By adjusting the levels of a key signaling protein, researchers improved motor function and brain abnormalities in experimental animals with a form of Huntington's disease, a severe neurodegenerative disorder. The new findings may lay the groundwork of a novel treatment for people with this fatal, progressive disease. "This research shows the intricate workings of a biological pathway crucial to the development of Huntington's disease, and is highly relevant to drug development," said study leader Beverly L. Davidson, Ph.D., director of The Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia (CHOP). "Our results in animals open the door to a promising potential therapy, based on carefully manipulating the dysregulated pathway to treat this devastating human disease." She added that restoring proper balance to these delicate biological processes may offer even broader benefits in treating other neurological diseases, such as amyotrophic lateral sclerosis (ALS), fragile X mental … Continue reading →