Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy.
Gentner B, Visigalli I, Hiramatsu H, Lechman E, Ungari S, Giustacchini A, Schira G, Amendola M, Quattrini A, Martino S, Orlacchio A, Dick JE, Biffi A, Naldini L.
Gentner B, et al. Among authors: amendola m.
Sci Transl Med. 2010 Nov 17;2(58):58ra84. doi: 10.1126/scitranslmed.3001522.
Sci Transl Med. 2010.
PMID: 21084719