GentnerB., VisigalliI., HiramatsuH., et al. (2010). Identification of hematopoietic stem cells-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci. Transl. Med., 2, 58ra84.
2.
LattanziA., NeriM., MadernaC., et al. (2010). Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models. Hum. Mol. Genet., 19, 2208–2227.
3.
VisigalliI., UngariS., MartinoS., et al. (2010). The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cells niche. Blood, 116, 1857–1866.
4.
BiffiA., AubourgP., and CartierN. (2011). Gene therapy for leukodystrophies. Hum. Mol. Genet., 20, R42–R53.
5.
BuchetD., GarciaC., DebouxC., et al. (2011). Human neural progenitors cells from different foetal forebrain regions remyelinate the adult mouse spinal cord. Brain, 134, 1168–1183.
NeriM., RiccaA., di GirolamoI., et al. (2011). Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy. Stem Cells, 29, 1559–1571.
8.
VisigalliI., and BiffiA. (2011). Maintenance of a functional hematopoietic stem cells niche through galactocerebrosidase and other enzymes. Curr. Opin. Hematol., 18, 214–219.
CapotondoA., MilazzoR., PolitiL.S., et al. (2012). Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cells transplantation. Proc. Natl. Acad. Sci. USA, 109, 15018–15023.
11.
ClarkeL.E., YoungK.M., HamiltonN.B., et al. (2012). Properties and fate of oligodendrocyte progenitor cells in the corpus callosum, motor cortex, and piriform cortex of the mouse. J. Neurosci., 32, 8173–8185.
12.
PiguetF., SondhiD., PiraudM., et al. (2012). Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice. Hum. Gene Ther., 23, 903–914.
13.
BiffiA., MontiniE., LorioliL., et al. (2013). Extensive genetic engineering of hematopoiesis with therapeutic benefit in metachromatic leukodystrophy patients after lentiviral hematopoietic stem cells gene therapy. Science, 341, 1233158.
14.
Krägeloh-MannI., GroeschelS., KehrerC., et al. (2013). Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort. Bone Marrow Transplant., 48, 369–375.
15.
MatsasR., and Baron-Van EvercoorenA. (2013). Glial cell transplantation. In Neuroglia, Section 3. Role of Glial Cells in Disease: Recovery of Neural Functions. KettenmanH. and RamsonB., ed. (Oxford University Press, New York, NY) pp. 728–745.
16.
RegisS., CorsoliniF., GrossiS., et al. (2013). Restoration of the normal splicing pattern of the PLP1 gene by means of an antisense oligonucleotide directed against an exonic mutation. PloS One, 8, e73633.
17.
von JonquieresG., MersmannN., KlugmannC.B., et al. (2013). Glial promoter selectivity following AAV-delivery to the immature brain. PLoS One, 8, e65646.