Gene Therapy Program

Beyond providing unmatched clinical care at Boston Children's, we're working around the clock to design approaches that let kids be kids. Gene therapy delivers DNA into a patient's cells to replace faulty or missing genes or adds new genes in an atte...

Beyond providing unmatched clinical care at Boston Children's, we're working around the clock to design approaches that let kids be kids. Gene therapy delivers DNA into a patient's cells to replace faulty or missing genes or adds new genes in an attempt to cure diseases or to make changes so the body is better able to fight off disease. The DNA for a gene or genes is carried into a patients cells by a delivery vehicle called a vector, typically a specially engineered virus. The vector then inserts the gene(s) into the cells' DNA.