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 UPIN Bonkowsky Lab Clement Lab Lai Lab Mei Lab Ghandehari Lab Our Impact 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) 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 AAV Mini-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy: 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
