Base Editing Hematopoietic Stem Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study

​ Conditions: CD40L-HyperIgM Syndrome

Interventions: Drug: Busulfan; Drug: Palifermin; Drug: Sirolimus; Drug: Alemtuzumab; Biological: Base-edited hematopoietic stem and progenitor cells

Sponsors: National Institute of Allergy and Infectious Diseases (NIAID)

Enrolling by invitation Conditions: CD40L-HyperIgM Syndrome
Interventions: Drug: Busulfan; Drug: Palifermin; Drug: Sirolimus; Drug: Alemtuzumab; Biological: Base-edited hematopoietic stem and progenitor cells
Sponsors: National Institute of Allergy and Infectious Diseases (NIAID)
Enrolling by invitation