When Jennie and Gary Landsman learned that their sons, Benny and Josh, had Canavan disease, a rare genetic neurological disorder characterized by the degeneration of white matter in the brain, they ...

Canavan disease is a rare, inherited leukodystrophy arising from mutations in the ASPA gene, which result in reduced activity of the enzyme aspartoacylase. This enzyme deficiency leads to the ...

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 ...

Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased brain myelin volume, resulting in promising functional ...