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Mouse Models of Metachromatic Leukodystrophy and Adrenoleukodystrophy

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Metachromatic leukodystrophy (MLD) and adrenoleukodystrophy (ALD) are two inherited �leukodystrophies that result in most cases in rapid destruction of the myelin within the central nervous system. There are no spontaneous animal models of these two leukodystrophies and knockout MLD and ALD mice have been generated by homologous recombination into murine embryonic stem cells. The initial analyses of these two mouse models were disappointing as they do not develop overt cerebral demyelination. Further and in-depth analysis has however revealed new and very important insights on the physiopathogenesis of MLD and ALD at an early stage of the disease. These data have now paved the way to new therapeutic approaches.
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