Structure–phenotype correlations of human CYP21A2 mutations in congenital adrenal hyperplasia
Structure–phenotype correlations of human CYP21A2 mutations in congenital adrenal hyperplasia
Mutations in the cytochrome p450 (CYP)21A2 gene, which encodes the enzyme steroid 21-hydroxylase, cause the majority of cases in congenital adrenal hyperplasia, an autosomal recessive disorder. To date, more than 100 CYP21A2 mutations have been reported. These mutations can be associated either with severe salt-wasting or simple virilizing phenotypes or with milder nonclassical phenotypes. Not all CYP21A2 mutations have, however, been characterized biochemically, and the clinical consequences of these mutations remain unknown. Using the crystal structure of its bovine homolog as a template, we have constructed a humanized model of CYP21A2 to provide comprehensive structural explanations for the clinical manifestations caused by each of the known disease-causing missense mutations in CYP21A2 . Mutations that affect membrane anchoring, disrupt heme and/or substrate binding, or impair stability of CYP21A2 cause complete loss of function and salt-wasting disease. In contrast, mutations altering the transmembrane region or conserved hydrophobic patches cause up to a 98% reduction in enzyme activity and simple virilizing disease. Mild nonclassical disease can result from interference in oxidoreductase interactions, salt-bridge and hydrogen-bonding networks, and nonconserved hydrophobic clusters. A simple in silico evaluation of previously uncharacterized gene mutations could, thus, potentially help predict the often diverse phenotypes of a monogenic disorder.
- Helmholtz Association of German Research Centres Germany
- Icahn School of Medicine at Mount Sinai United States
- Queen's University Belfast United Kingdom
- National Research Council Italy
- Institute for Complex Systems Italy
Models, Molecular, Adrenal Hyperplasia, Congenital, Protein Conformation, Cell Membrane, Mutation, Missense, 610, Computational Biology, Hydrogen Bonding, Heme, Molecular Dynamics Simulation, Phenotype, Animals, Humans, Cattle, Steroid 21-Hydroxylase, Protein Binding
Models, Molecular, Adrenal Hyperplasia, Congenital, Protein Conformation, Cell Membrane, Mutation, Missense, 610, Computational Biology, Hydrogen Bonding, Heme, Molecular Dynamics Simulation, Phenotype, Animals, Humans, Cattle, Steroid 21-Hydroxylase, Protein Binding
78 Research products, page 1 of 8
- 2017IsRelatedTo
- 2020IsAmongTopNSimilarDocuments
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2020IsAmongTopNSimilarDocuments
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
chevron_left - 1
- 2
- 3
- 4
- 5
chevron_right
citations This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).119 popularity This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.Top 10% influence This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).Top 10% impulse This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.Top 1%
