Duchenne Muscular Dystrophy

Data from L.R. Rodinoklapac and colleagues advance knowledge in duchenne muscular dystrophy

Published in Gene Therapy Weekly, April 8th, 2010

"Animal models for Duchenne muscular dystrophy (DMD) have species limitations related to assessing function, immune response, and distribution of micro- or mini-dystrophins. Nonhuman primates (NHPs) provide the ideal model to optimize vector delivery across a vascular barrier and provide accurate dose estimates for widespread transduction," investigators in the United States report.

"To address vascular delivery and dosing in rhesus macaques, we have generated a fusion construct that encodes an eight amino-acid FLAG epitope at the C-terminus of micro-dystrophin to facilitate translational studies targeting DMD. Intramuscular (IM) injection of AAV8. MCK. micro-dys. FLAG...

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Source: Gene Therapy Weekly (2010-04-08)

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