Therapeutics: gene therapy

We are working with Dr. Steven Gray from UT Southwestern to develop a gene therapy for DHPS Deficiency. Dr. Gray has developed several previous gene therapies that are currently in clinical trials in patients with other neurogenetic conditions. The DHPS gene therapy is currently being evaluated in mice to determine safety and efficacy. We hope to advance this gene therapy to clinical trials to supply patients with DHPS Deficiency with a healthy copy of the gene.