First Description of the Generation of Human Schwann Cells Transfected With CRISPR Cas9 and a Model of Metformin Mitochondrial Metabolism in Metachromatic Leukodystrophy
Abstract:Background: Metachromatic leukodystrophy (MLD) is a neurological lysosomal deposit disease that has an impact on public health despite its low incidence in the population. Existing treatments are expensive and inefficient. Few reports in the literature on pathophysiological events related to enzyme deficiency and subsequent accumulation of sulfatides; therefore, the use of metformin as an alternative treatment was evaluated in vitro to counteract the effects. Methodology: An experimental in vitro study that so… Show more
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