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Multi-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapy.

PLoS ONE. 2015; 
ParkJoshua,WickiJacqueline,KnoblaughSue E,ChamberlainJeffrey S,LeeDong
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Custom Vector Construction … All mice were euthanized following the conclusion of the study Generation of Constructs, Vector Production and Delivery A codon-optimized version of micro-dystrophin (ΔR4-23/ΔCT) cDNA [35] was designed and synthesized by GenScript (Piscataway, NJ) … Get A Quote

摘要

The objective of this study was to investigate the efficacy of using quantitative magnetic resonance imaging (MRI) as a non-invasive tool for the monitoring of gene therapy for muscular dystrophy. The clinical investigations for this family of diseases often involve surgical biopsy which limits the amount of information that can be obtained due to the invasive nature of the procedure. Thus, other non-invasive tools may provide more opportunities for disease assessment and treatment responses. In order to explore this, dystrophic mdx4cv mice were systemically treated with a recombinant adeno-associated viral (AAV) vector containing a codon-optimized micro-dystrophin gene. Multi-parametric MRI of T2, magnet... More

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