Outcomes of Umbilical Cord Blood Transplantation in Children with Batten Disease

摘要

Batten Disease (BD) is an inherited metabolic disease caused byvarious mutations in ceroid lipofuscinoses (CLN) genes. Symptomsvary and include vision loss, behavioral changes, loss of motorand/or cognitive skills, and seizures. Death typically occurs inchildhood or adolescence depending on the gene. Umbilical cordblood transplant (UCBT) is effective in treating many inheritedmetabolic diseases with limited experience in BD.