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Updated: Aug 20, 2026
Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
[Study on the recombinant adeno-associated virus vector carrying LacZ gene expression in the skeletal muscle]
Hong-hua Li1, Su-ming Zhang, Si-yu Fang
1Department of Neurology, Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, Hubei, 430030 P. R. China.
Objective:
To look for a gene delivery route to the treatment of Duchenne muscular dystrophy(DMD).
Methods:
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.
Results:
(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.
Conclusion:
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.
