Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy

Vasco Meneghini, Annalisa Lattanzi, Luigi Tiradani, Gabriele Bravo, Francesco Morena, Francesca Sanvito, Andrea Calabria, John Bringas, Jeanne M. Fisher-Perkins, Jason P. Dufour, Kate C. Baker, Claudio Doglioni, Eugenio Montini, Bruce A. Bunnell, Krystof Bankiewicz, Sabata Martino, Luigi Naldini, Angela Gritti

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Medicine & Life Sciences