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Home » Locus-specific editing of the 21-hydroxylase gene restores adrenal function in a CAH model
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Endocrine/metabolic

Locus-specific editing of the 21-hydroxylase gene restores adrenal function in a CAH model

Nov. 26, 2025
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Glucocorticoid replacement therapy is the current standard of care for congenital adrenal hyperplasia (CAH). However, new therapeutic strategies that can better recapitulate physiological requirements and reduce morbidity and mortality among CAH patients are urgently needed. Despite the promise of gene therapy for correcting monogenic disorders, the strategies investigated to date have not yielded satisfactory results.
BioWorld Science Endocrine/metabolic

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