U.S., May 8 -- ClinicalTrials.gov registry received information related to the study (NCT06959771) titled 'Base Editing Hematopoietic Stem Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study' on May 06.
Brief Summary: Background:
X-linked Hyper IgM (HIGM) syndrome is caused by a mutation in the CD40L gene. People with this disease have white blood cells that do not work properly. These people are at risk of severe infections and autoimmune diseases. Stem cell transplant can cure this disease. However, transplanting stem cells donated by other people can have serious complications. Another approach is gene therapy; this treatment repairs the mutation in a person's own stem cells by base editing. Researchers want to know if ...