Published in J Clin Invest on April 01, 2004
Gene Therapy for Metachromatic Leukodystrophy (TIGET-MLD) | NCT01560182
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Friend or Foe? Resident Microglia vs Bone Marrow-Derived Microglia and Their Roles in the Retinal Degeneration. Mol Neurobiol (2016) 0.76
Haematopoietic stem cell transplantation does not retard disease progression in the psycho-cognitive variant of late-onset metachromatic leukodystrophy. J Inherit Metab Dis (2010) 0.76
Lentivector integration sites in ependymal cells from a model of metachromatic leukodystrophy: non-B DNA as a new factor influencing integration. Mol Ther Nucleic Acids (2014) 0.76
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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science (2013) 5.34
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Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. J Clin Invest (2006) 1.54
Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood (2004) 1.53
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