Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle

Alessandra Tessitore, Armida Faella, Thomas O'Malley, Gabriella Cotugno, Monica Doria, Tetsuo Kunieda, Giuseppe Matarese, Mark Haskins, Alberto Auricchio

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33 Citations (Scopus)

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