Abstract
Campomelic dysplasia (CD, MIM 114290) is a rare, often lethal, dominantly inherited, congenital skeletal dysplasia, associated with male-to-female autosomal sex reversal and due to de novo mutations of the SOX9 gene, a tissue-specific transcription factor gene involved both in skeletogenesis and male sexual differentiation. Here we report on a 4 months-old 46,XY sex reversed infant with typical clinical features for CD due to a novel mutation of the SOX9 gene, Q401X, leading to synthesis of a truncated SOX9 protein that completely lacks the C-terminal transactivation domain.
MeSH terms
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Abnormalities, Multiple / diagnosis
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Abnormalities, Multiple / genetics*
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Campomelic Dysplasia / diagnosis
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Campomelic Dysplasia / genetics*
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Chromosome Aberrations*
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Codon, Nonsense / genetics*
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DNA Mutational Analysis
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Fatal Outcome
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Female
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Genetic Carrier Screening
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Gonadal Dysgenesis, 46,XY / diagnosis
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Gonadal Dysgenesis, 46,XY / genetics*
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Humans
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Infant
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Infant, Newborn
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Karyotyping
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Male
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Pregnancy
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Respiratory Distress Syndrome, Newborn / diagnosis
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Respiratory Distress Syndrome, Newborn / genetics
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SOX9 Transcription Factor / genetics*
Substances
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Codon, Nonsense
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SOX9 Transcription Factor
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SOX9 protein, human