Overexpression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycoprotein/polylysine/DNA complexes.

@article{Stankovics1994OverexpressionOH,
  title={Overexpression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycoprotein/polylysine/DNA complexes.},
  author={J. Stankovics and Ana Maria Crane and Emmet Andrews and Catherine H. Wu and Gary Wu and Fred David Ledley},
  journal={Human gene therapy},
  year={1994},
  volume={5 9},
  pages={1095-104}
}
Methylmalonic acidemia resulting from genetic deficiency of methylmalonyl CoA mutase (MCM) is an often fatal metabolic disease. Somatic gene therapy for this disorder may require gene replacement in the liver. We describe overexpression of MCM in the liver of mice after in vivo gene delivery using asialoglycoprotein/polylysine/DNA (ASO/PL/DNA) targeted delivery to the liver of plasmids expressing recombinant MCM. After intravenous administration of the ASO/PL/DNA complex, the vector sequences… CONTINUE READING

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