Gene Therapy for Sickle Cell Disease An unprespecified in | UPDATES IN MEDICINE
Gene Therapy for Sickle Cell Disease
An unprespecified interim analysis of results from a phase 1–2 study of gene therapy for sickle cell disease shows resolution of severe vaso-occlusive events in 25 patients who could be evaluated. In the 24 months before enrollment, these patients had a median of 3.5 severe vaso-occlusive events per year.
