An AAVrh10-CAG-CYP21-HA vector allows persistent correction of 21-hydroxylase deficiency in a Cyp21−/− mouse model
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DOI: 10.1038/gt.2017.10
Abstract: The treatment of severe forms of 21-hydroxylase deficiency (21OHD) remains unsatisfactory in many respects. As a monogenic disease caused by loss-of-function mutations, 21OHD is a potential candidate for a gene therapy (GT) approach. The first… read more here.
Keywords: vector; cyp21; hydroxylase deficiency; cyp21 vector ... See more keywords