103. Development of an effective and safe hematopoietic stem cell gene therapy for globoid cell leukodystrophy: The unexpected issue of GALC toxicity

Visigalli, Ilaria; Cesani, Martina; Neri, Margherita; Ungari, Silvia; Tononi, Laura; Marchesini, Sergio; Grez, Manuel; Gritti, Angela; Naldini, Luigi; Biffi, Alessandra

doi:10.1016/j.ymgme.2007.10.115

Molecular Genetics and Metabolism

2008

DOI: 10.1016/j.ymgme.2007.10.115

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103. Development of an effective and safe hematopoietic stem cell gene therapy for globoid cell leukodystrophy: The unexpected issue of GALC toxicity

Ilaria Visigalli

Martina Cesani

Margherita Neri

et al.

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103. Development of an effective and safe hematopoietic stem cell gene therapy for globoid cell leukodystrophy: The unexpected issue of GALC toxicity

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