TY  - GEN
AU  - Gentner,Bernhard
AU  - Visigalli,Ilaria
AU  - Hiramatsu,Hidefumi
AU  - Lechman,Eric
AU  - Ungari,Silvia
AU  - Giustacchini,Alice
AU  - Schira,Giulia
AU  - Amendola,Mario
AU  - Quattrini,Angelo
AU  - Martino,Sabata
AU  - Orlacchio,Aldo
AU  - Dick,John E
AU  - Biffi,Alessandra
AU  - Naldini,Luigi
TI  - Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
SN  - 1946-6242
PY  - 2011///0610
KW  - Animals
KW  - Cell Differentiation
KW  - Cell Separation
KW  - Cytoprotection
KW  - Galactosylceramidase
KW  - metabolism
KW  - Gene Expression Regulation
KW  - Genes, Transgenic, Suicide
KW  - Genetic Therapy
KW  - Hematopoietic Stem Cells
KW  - cytology
KW  - Humans
KW  - Leukodystrophy, Globoid Cell
KW  - genetics
KW  - Mice
KW  - Mice, SCID
KW  - MicroRNAs
KW  - Organ Specificity
N1  - Publication Type: Journal Article; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1126/scitranslmed.3001522
ER  -