Genetic research
Gene Therapy for Hemophilia: From Basic Science to First Approvals of "One-and-Done" Therapies
Research conducted at the Indiana University School of Medicine has led to significant advancements in gene therapy for hemophilia. The study, published in Molecular Therapy, highlighted the emergence of AAV gene transfer to hepatocytes as a promising strategy for treating hemophilia A and B. Funded by the National Institutes of