Insulin-like growth factor-1 and neurotrophin-3 gene therapy prevents motor decline in an X-linked adrenoleukodystrophy mouse model

Mastroeni, RobertoBensadoun, Jean-CharlesCharvin, DelphineAebischer, PatrickPujol, AuroraRaoul, Cédric2009-10-142009-10-142009-10-14200910.1002/ana.21677https://infoscience.epfl.ch/handle/20.500.14299/43684WOS:000268847600019X-linked adrenoleukodystrophy (X-ALD) is the most common inherited peroxisomal disorder characterized by a progressive demyelination of the central nervous system. The marked loss of myelin and oligodendrocytes observed in the disease prompted us to evaluate the therapeutic potential of insulin-like growth factor-1 and neurotrophin-3, two potent inducers of myelin formation and oligodendrocyte survival. Viral vectors engineered to produce insulin-like growth factor-1 or neurotrophin-3 were administrated into the cerebrospinal fluid of an X-linked adrenoleukodystrophy mouse model. We show that viral-based, long-lasting delivery of insulin-like growth factor-1 and neurotrophin-3 significantly halts the progression of the disease and leads to potent protective effect against the demyelination processCentral-Nervous-SystemEnhances RemyelinationRna InterferenceDeliveryMiceCellsOnsetDemyelinationInactivationProgressionInsulin-like growth factor-1 and neurotrophin-3 gene therapy prevents motor decline in an X-linked adrenoleukodystrophy mouse modeltext::journal::journal article::research article