Home Pediatrics Pediatrics Research Programs Center for Gene Therapy Posters & Publications Home Pediatrics Pediatrics Research Programs Center for Gene Therapy Posters & Publications Close Pediatrics Research Department of Research Back to Center for Gene Therapy Meet Our Team Posters & Publications Gene Therapy Research Research Bonkowsky Lab UPIN Communications Conference Posters ASGCT 2025 AAV Delivery of ADAR Mediated RNA Editing for Treatment of Collagen VI-Related Muscular Dystrophy Bonkowsky Lab - AAV-Mediated Gene Supplementation in Combination with Microglial Replacement for Vanishing White Matter Disease Using Circulation-Derived Myeloid Cells (CDMCs) AAV Mini-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy: a Phase 1b Trial Implementation of an Institutional Gene Therapy Safety Committee at the University of Utah Publications 2025 Complement Activation in a Phase Ib Study of Fordadistrogene Movaparvovec for Duchenne Muscular Dystrophy Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy Initial observations from a phase 1b trial Delandistrogene Moxeparvovec Gene Therapy in Individuals with Duchenne Muscular Dystrophy: Evidence in Focus Institutional readiness for novel therapeutics: A framework for multidisciplinary integration 2024 Taking ACTION to detect myocarditis related to recombinant gene transfer therapy for Duchenne Muscular Dystrophy; Consensus recommendations for cardiac surveillance Media Pieces 2024 Beacon of Hope - Gold Award for Excellence from AAMC
