Sharing Approaches for Splice Regulation & Modular Protein Function in Retinal Gene Therapy

Time: 2:05 pm
day: Day One Track A - Afternoon


  • Gene therapy of the alternatively spliced gene RPGR in X-linked retinitis pigmentosa may be influenced by stringently regulator splicing of its alternative isoforms
  • Modular protein function assists in designing efficacious gene therapy for large genes that are not packageable into AAV vectors