Study maps enzyme target for potential treatment of rare genetic disorder

https://www.news-medical.net/news/20261113/Study-maps-enzyme-target-for-potential-treatment-of-rare-genetic-disorder.aspx Researchers at the Icahn School of Medicine at Mount Sinai have mapped an enzyme that could be targeted to develop new treatments for glutaric aciduria type 1 (GA1), a rare genetic disorder that can lead to brain damage, revealing new details about how the enzyme works and how its activity is controlled. The findings were published in the November 13 online issue of Science Advances