[PDF][PDF] Mandibuloacral dysplasia is caused by a mutation in LMNA-encoding lamin A/C

G Novelli, A Muchir, F Sangiuolo… - The American Journal of …, 2002 - cell.com
The American Journal of Human Genetics, 2002cell.com
Mandibuloacral dysplasia (MAD) is a rare autosomal recessive disorder, characterized by
postnatal growth retardation, craniofacial anomalies, skeletal malformations, and mottled
cutaneous pigmentation. The LMNA gene encoding two nuclear envelope proteins (lamins
A and C [lamin A/C]) maps to chromosome 1q21 and has been associated with five distinct
pathologies, including Dunnigan-type familial partial lipodystrophy, a condition that is
characterized by subcutaneous fat loss and is invariably associated with insulin resistance …
Mandibuloacral dysplasia (MAD) is a rare autosomal recessive disorder, characterized by postnatal growth retardation, craniofacial anomalies, skeletal malformations, and mottled cutaneous pigmentation. The LMNA gene encoding two nuclear envelope proteins (lamins A and C [lamin A/C]) maps to chromosome 1q21 and has been associated with five distinct pathologies, including Dunnigan-type familial partial lipodystrophy, a condition that is characterized by subcutaneous fat loss and is invariably associated with insulin resistance and diabetes. Since patients with MAD frequently have partial lipodystrophy and insulin resistance, we hypothesized that the disease may be caused by mutations in the LMNA gene. We analyzed five consanguineous Italian families and demonstrated linkage of MAD to chromosome 1q21, by use of homozygosity mapping. We then sequenced the LMNA gene and identified a homozygous missense mutation (R527H) that was shared by all affected patients. Patient skin fibroblasts showed nuclei that presented abnormal lamin A/C distribution and a dysmorphic envelope, thus demonstrating the pathogenic effect of the R527H LMNA mutation.
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