Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

Alessandra Biffi, Alessia Capotondo, Stefania Fasano, Ubaldo Del Carro, Sergio Marchesini, Hisaya Azuma, Maria Chiara Malaguti, Stefano Amadio, Riccardo Brambilla, Markus Grompe, Claudio Bordignon, Angelo Quattrini, Luigi Naldini

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Medicine & Life Sciences