Gene Therapy Breakthrough for Rare Genetic Disorder
In a historic medical breakthrough, doctors have successfully used gene-editing therapy to treat an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. The rare genetic disorder affects one in 1.3 million babies and can be fatal within the first week of life, with survivors suffering from severe mental and developmental delays. KJ Muldoon, … Read more