The first peer-reviewed journal in the field of human gene therapy, providing all-inclusive coverage of the research, methods, and clinical developments that are driving today's explosion of gene ...

Scientists at City of Hope have developed universal donor stem cells that could one day provide lifesaving therapy to children with lethal brain conditions, such as Canavan disease, as well as to ...

- Following treatment, the N-acetylaspartate (NAA) levels of CANaspire participants are consistent with levels seen in individuals with milder Canavan disease based on findings from the Company’s ...