Improving gene therapy approaches for globoid cell leukodystrophy: pre-clinical testing of chimeric GALC enzymes with enhanced bioavailability in human neural and hematopoietic cells

Improving gene therapy approaches for globoid cell leukodystrophy: pre-clinical testing of chimeric GALC enzymes with enhanced bioavailability in human neural and hematopoietic cells / Ricca, A; Freschi, M; Valeri, E; Unali, G; Cascino, F; Morena, F; Martino, S; Kajaste-Rudnitski, A; Gritti, A. - In: HUMAN GENE THERAPY. - ISSN 1043-0342. - 33:23-24(2022). ( ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022) [10.1089/hum.2022.29225.abstracts].