In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

Nature Medicine - Tập 7 Số 3 - Trang 310-316 - 2001
Antonella Consiglio1, Angelo Quattrini1, Sabata Martino1, Jean Charles Bensadoun2, Diego Dolcetta1, A. Trojani1, Giuliana Benaglia3, Sergio Marchesini3, Vincenzo Cestari4, Alberto Oliverio4, Claudio Bordignon1, Luigi Naldini5
1Telethon Institute for Gene Therapy and Department of Neurology, Scientific Institute H.S. Raffaele HSR-TIGET, Milan, Italy.
2Division of Surgical Research and Gene Therapy Center, Lausanne, Switzerland
3Department of Biomedical Science and Biotechnology, University of Brescia, Brescia, Italy
4Institute of Psychobiology and Psychopharmacology (CNR), Rome, Italy
5Laboratory for Gene Transfer and Therapy, Institute for Cancer Research and Treatment, University of Torino, Torino, Italy

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