Full correction of neurologic and somatic lysosomal pathology in Mucopolysaccharidosis type IIIB by AAV9-based gene therapy

V Haurigot, A Ribera, S Marco, M Garcia, S Motas, P Villacampa, V Sanchez, L Maggioni, C Roca, X Leon, M Molas, Munoz S, J Ruberte, F Mingozzi, X Moll, S Anor, F. Bosch

Research output: Contribution to journalArticleResearch

Original languageEnglish
Pages (from-to)A37-
JournalHuman Gene Therapy
Issue number11
Publication statusPublished - 1 Jan 2014

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