Pathogenic variant in NFIX gene affecting three sisters due to paternal mosaicism.
Nydia Rena Benita SihombingTri Indah WinarniHans van BokhovenIneke van der BurgtNicole de LeeuwSultana Muhammad Hussein FaradzPublished in: American journal of medical genetics. Part A (2020)
We present a family with three girls presenting similar dysmorphic features, including overgrowth, intellectual disability, macrocephaly, prominent forehead, midface retrusion, strabismus, and scoliosis. Both parents were unaffected, suggesting the presence of an autosomal recessive syndrome. Following exome sequencing, a heterozygous nonsense variant was identified in the NFIX gene in all three siblings. The father appeared to have a low-grade (7%) mosaicism for this variant in his blood. Previously, de novo pathogenic variants in NFIX have been identified in Marshall-Smith syndrome and Malan syndrome, which share distinctive phenotypic features shared with the patients of the present family. This case emphasizes the importance of further molecular analysis especially in familial cases, to exclude the possibility of parental mosaicism.