Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs

@inproceedings{Hakim2014SystemicGT,
  title={Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs},
  author={Chady H. Hakim and Yongping Yue and Jin-Hong Shin and Regina R. Williams and Keqing Zhang and Bruce F. Smith and Dongsheng Duan},
  booktitle={Molecular therapy. Methods & clinical development},
  year={2014}
}
The muscular dystrophies are a group of devastating genetic disorders that affect both skeletal and cardiac muscle. An effective gene therapy for these diseases requires bodywide muscle delivery. Tyrosine mutant adeno-associated virus (AAV) has been considered as a class of highly potent gene transfer vectors. Here, we tested the hypothesis that systemic… CONTINUE READING