期刊
ANNALS OF NEUROLOGY
卷 66, 期 1, 页码 117-122出版社
WILEY
DOI: 10.1002/ana.21677
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资金
- ICREA Funding Source: Custom
X-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 oligodendroctes observed in the disease prompted us to evaluate the therapeutic potential Of Insulinlike growth factor-l 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 process.
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