Gene Therapy Strategy Restores Motor Learning in Early Mouse Study

Gene Therapy Strategy Restores Motor Learning in Early Mouse Study

313685

Gene Therapy Strategy Restores Motor Learning in Early Mouse Study

A novel dual gene therapy rescued the function of the UBE3A gene in a mouse model of Angelman syndrome, the gene that is faulty in people with the condition, and restored motor-skill learning and essential behaviors, a study has demonstrated. “This was a proof-of-concept study, but if these early results were translated to the clinic, they would represent big improvements in the quality of life for individuals with Angelman syndrome,” Matt Judson, PhD, from the…

You must be logged in to read/download the full post.