biostudies-literatureTaghian TUniversity of Massachusetts Medical SchoolNorthwestern UniversityNational Institutes of HealthNIGMS NIH HHSNational Science Foundation128-134https://www.ebi.ac.uk/biostudies/studies/S-EPMC8517204biostudies-literatureUnknown23Transformative results of adeno-associated virus (AAV) gene therapy in patients with spinal muscular atrophy and Leber's congenital amaurosis led to approval of the first two AAV products in the United States to treat these diseases. These extraordinary results led to a dramatic increase in the number and type of AAV gene-therapy programs. However, the field lacks non-invasive means to assess levels and duration of therapeutic protein function in patients. Here, we describe a new magnetic resonance imaging (MRI) technology for real-time reporting of gene-therapy products in the living animal in the form of an MRI probe that is activated in the presence of therapeutic protein expression. For the first time, we show reliable tracking of enzyme expression after a now in-human clinical trial AMolecular therapy. Methods & clinical developmentReal-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis.PMC8517204T32 GM00838210R01EB005866-08T32 GM105538Li HBatista ARZheng STaghian TLilley LMartin DRMeade TJTodeasa SGray-Edwards HLGounis MJCaldwell MMaguire AKamper SKing RMSena-Esteves MMesa KRodriguez PfalseReal-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis.Transformative results of adeno-associated virus (AAV) gene therapy in patients with spinal muscular atrophy and Leber's congenital amaurosis led to approval of the first two AAV products in the United States to treat these diseases. These extraordinary results led to a dramatic increase in the number and type of AAV gene-therapy programs. However, the field lacks non-invasive means to assess levels and duration of therapeutic protein function in patients. Here, we describe a new magnetic resonance imaging (MRI) technology for real-time reporting of gene-therapy products in the living animal in the form of an MRI probe that is activated in the presence of therapeutic protein expression. For the first time, we show reliable tracking of enzyme expression after a now in-human clinical trial A2021-01-01T00:00:00Z2021 Dec2026-05-07T22:20:31.969Z2025-02-19T03:53:29.995ZS-EPMC85172043470383610.1016/j.omtm.2021.08.003