Research ArticleGloboid Cell Leukodystrophy

Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

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Science Translational Medicine  17 Nov 2010:
Vol. 2, Issue 58, pp. 58ra84
DOI: 10.1126/scitranslmed.3001522

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