Lentiviral-mediated correction of MPS VI cells and gene transfer to joint tissues.

Abstract

Joint disease in mucopolysaccharidosis type VI (MPS VI) remains difficult to treat despite the success of enzyme replacement therapy in treating other symptoms. In this study, the efficacy of a lentiviral vector to transduce joint tissues and express N-acetylgalactosamine-4-sulphatase (4S), the enzyme deficient in MPS VI, was evaluated in vitro and the… (More)
DOI: 10.1016/j.ymgme.2009.02.008

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