Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy.

@article{Grimm2003PreclinicalIV,
  title={Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy.},
  author={Dirk Grimm and Shangzhen Zhou and Hiroyuki Nakai and Clare E. Thomas and Theresa A Storm and Sally Fuess and Takashi Matsushita and James E. Allen and Richard T Surosky and Michael A Lochrie and Leonard A Meuse and Alan McClelland and Peter Colosi and Mark A. Kay},
  journal={Blood},
  year={2003},
  volume={102 7},
  pages={2412-9}
}
We report the generation and use of pseudotyped adeno-associated viral (AAV) vectors for the liver-specific expression of human blood coagulation factor IX (hFIX). Therefore, an AAV-2 genome encoding the hfIX gene was cross-packaged into capsids of AAV types 1 to 6 using efficient, large-scale technology for particle production and purification. In immunocompetent mice, the resultant vector particles expressed high hFIX levels ranging from 36% (AAV-4) to more than 2000% of normal (AAV-1, -2… CONTINUE READING