In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy.
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DOI: 10.1007/978-1-4939-8651-4_27
Abstract: Spinal muscular atrophy (SMA), the most common gentic cause of infantile death caused by mutations in the SMN1 gene, presents a unique case in the field of splice modulation therapy, where a gene (or lack… read more here.
Keywords: evaluation antisense; spinal muscular; vitro evaluation; muscular atrophy ... See more keywords