While the room was abuzz with excitement that day, “he slept through the entire thing,” recalled study author Dr. Rebecca Ahrens-Nicklas, a gene therapy expert at CHOP. After follow-up doses ...

GEMMABio was founded by gene therapy scientist Jim Wilson who led an influential research group at the University of Pennsylvania. The group developed methods for delivering genes to cells, which ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root ...

I am excited about the research in gene therapy for hemophilia.1 I have lived with hemophilia A for 57 years; however, my bleeding episodes are not as severe as Don Miller's. I have worked in ...

Y.P. Yang, F.A. Nunes, K. Berencsi, E. Gonczol, J.F. Engelhardt, J.M. Wilson, "Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis," Nature ...