OR25-01 Durable CYP21A2 Gene Therapy in Non-Human Primates for Treatment of Congenital Adrenal Hyperplasia

Severe Congenital Adrenal Hyperplasia (CAH) is most commonly caused by genetic defects in the CYP21A2 gene, which leads to a deficiency of 21-hydroxylase enzyme and disruption in the biosynthesis of Adrenal corticosteriods. Despite treatment with corticosteroids, patients remain at significant risk...

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Detalles Bibliográficos
Autores principales: Eclov, Rachel J, Lewis, Terra E W, Kapadia, Mayank, Scott, David W, McCoy, Daniel D, Rouse, Jeremy L, Romero, Kirsten B, Beard, Clayton W, Mansfield, Gary S
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
Adrenal
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7209390/
http://dx.doi.org/10.1210/jendso/bvaa046.899

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