Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis
Toloo Taghian, Ana Rita Batista, Sarah Kamper, Michael A. Caldwell, Laura M. Lilley, Hao Li, Paola Rodrı́guez, Katerina Mesa, Shaokuan Zheng, R King, Matthew J. Gounis, Sophia H. Todeasa, Anne S. Maguire, Douglas R. Martin, Miguel Sena‐Esteves, Thomas J. Meade, Heather Gray‐Edwards
Abstract
) correlates with βgal activity in central nervous system and peripheral organs after intracranial or intravenous AAV gene therapy, respectively. With >1,800 gene therapies in phase I/II clinical trials (ClinicalTrials.gov), development of a non-invasive method to track gene expression over time in patients is crucial to the future of the gene-therapy field.
Topics & Concepts
Genetic enhancementMedicineMagnetic resonance imagingGene expressionSpinal muscular atrophyAdeno-associated virusGeneGene deliveryPathologyBioinformaticsBiologyDiseaseRecombinant DNAVector (molecular biology)RadiologyGeneticsNeurogenetic and Muscular Disorders ResearchVirus-based gene therapy researchRNA Interference and Gene Delivery