[HTML][HTML] A mutation in the thyroid hormone receptor alpha gene

E Bochukova, N Schoenmakers… - … England Journal of …, 2012 - Mass Medical Soc
E Bochukova, N Schoenmakers, M Agostini, E Schoenmakers, O Rajanayagam, JM Keogh…
New England Journal of Medicine, 2012Mass Medical Soc
Thyroid hormones exert their effects through alpha (TRα1) and beta (TRβ1 and TRβ2)
receptors. Here we describe a child with classic features of hypothyroidism (growth
retardation, developmental retardation, skeletal dysplasia, and severe constipation) but only
borderline-abnormal thyroid hormone levels. Using whole-exome sequencing, we identified
a de novo heterozygous nonsense mutation in a gene encoding thyroid hormone receptor
alpha (THRA) and generating a mutant protein that inhibits wild-type receptor action in a …
Thyroid hormones exert their effects through alpha (TRα1) and beta (TRβ1 and TRβ2) receptors. Here we describe a child with classic features of hypothyroidism (growth retardation, developmental retardation, skeletal dysplasia, and severe constipation) but only borderline-abnormal thyroid hormone levels. Using whole-exome sequencing, we identified a de novo heterozygous nonsense mutation in a gene encoding thyroid hormone receptor alpha (THRA) and generating a mutant protein that inhibits wild-type receptor action in a dominant negative manner. Our observations are consistent with defective human TRα-mediated thyroid hormone resistance and substantiate the concept of hormone action through distinct receptor subtypes in different target tissues.
The New England Journal Of Medicine