Gene & Cell TherapyAAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich Ataxia: A Nonrandomized Clinical TrialRGRonald G CrystalJWJonathan W WeinsaftSMStephen M. KaminskyRJRobert J. KanerASAbraham Sanders+4 more →JAMA cardiology · 2026
Gene & Cell TherapySlowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2DSDolan SondhiSMStephen M. KaminskyNRNeil R. HackettOPOdelya Pagovich+11 more →RGRonald G CrystalScience translational medicine · 2020
Gene & Cell TherapyQuantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman PrimatesDJDouglas J BallonEKEdward K FungLALinda A HeierDSDolan Sondhi+1 more →RGRonald G CrystalHuman gene therapy · 2020