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Gene Therapy for Neuropathies

principal investigator : Assumpció Bosch assumpcio.bosch@uab.cat Department: Bioquímica i Biologia Molecular, Unitat de Biociències Center: CBATEG Institution: UAB (Universitat Autònoma de Barcelona) Members: 5 Research lines: 1 - Gene therapy for neurological complications of diabetes. 2 - Study of the mechanisms that lead to neurodegeneration of mucopolysaccharidosis 3 - Gene therapy for type VII mucopolysaccharidosis. Areas:  Central Nervous System Disorders   Neuronal death  Keywords: Gene therapy, neurodegeneration, Mucopolysaccharidosis, diabetic neuropathy, AAV vectors, non-human adenoviral vectors, lentiviral vectors. Publications: J. Homs, G. Pagès, L. Ariza, C. Casas, M. Chillon, X. Navarro, A. Bosch (2014). Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy. Molecular Therapy-Methods and Clinical Development 1, 7; doi:10.1038/mtm.2013.7 L. Ariza, L. Giménez-Llort, A. Cubizolle, G. Pagès, B. Garcia-Lareu, N. Serratrice, D. Cots, R. Thwaite, M. Chillon, E.J. Kremer, A. Bosch (2013). CNS delivery of helper-dependent canine adenovirus corrects neuropathology and behavior in Mucopolysaccharidosis type VII mice. Human Gene Therapy, in press. PMID:24299455  J. Homs, L. Ariza, G. Pagès, E. Udina, X. Navarro, M. Chillón, A. Bosch (2011). Schwann cell targeting via intrasciatic injection of AAV8 as gene therapy strategy for peripheral nerve regeneration. Gene Therapy 18(6):622-30 Group Web site