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Publication 12/01/2014

Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM

Molecular Therapy : The Journal Of The American Society Of Gene Therapy

Publication 10/14/2014

Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.

Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM

Proceedings Of The National Academy Of Sciences Of The United States Of America