Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5
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DOI: 10.1038/s41467-020-16605-x
Abstract: Currently, there are no treatments for Alport syndrome, which is the second most commonly inherited kidney disease. Here we report the development of an exon-skipping therapy using an antisense-oligonucleotide (ASO) for severe male X-linked Alport… read more here.
Keywords: exon skipping; linked alport; alport syndrome; skipping therapy ... See more keywords