Study on the recombinant adeno-associated virus vector carrying LacZ gene expression in the skeletal muscle / 中华医学遗传学杂志

ABSTRACT

<p><b>OBJECTIVE</b>To look for a gene delivery route to the treatment of Duchenne muscular dystrophy(DMD).</p><p><b>METHODS</b>The recombinant adeno-associated virus vector(rAAV) carrying a LacZ reporter gene was constructed. rAAVLacZ was delivered into the skeletal muscle tissue of C57/BL6 mice by intramuscular injection. Then an intraarterial delivery route was taken to reveal whether rAAVLacZ could transduce muscle tissue.</p><p><b>RESULTS</b>(1) The LacZ gene was efficiently transduced and expressed persisting for 5 months after intramuscular injection. (2) The membrane of muscle and smooth muscle of vessel was widely transduced by intra-arterial delivery rAAVLacZ.</p><p><b>CONCLUSION</b>These data provide the evidence that rAAVLacZ can efficiently transduce muscle for a long period. Improving intraarterial gene delivery will be promising means for rAAV-mediated gene therapy for generalized skeletal muscle of DMD.</p>