New Gene Therapy Treatments: Presented by NBDF and HTRS скачать в хорошем качестве

New Gene Therapy Treatments: Presented by NBDF and HTRS

This session of the Research Journal Club will focus on gene therapy. Gene therapy for hemophilia A is now commercially available in some countries. While early patient data show a modest reduction in disease severity, current FVIII gene therapies using viral delivery have not yet maintained consistent desired results year after year. This session will explore current work in Dr. Lindsey George’s laboratory at the Children’s Hospital of Philadelphia to use mechanisms of FVIIIa inactivation to engineer enhanced transgenes for hemophilia A gene therapy. Speakers: Lindsey George, MD; John Morris, PhD; Bill Maurits