Two novel mutations in TBC1D32 add complexity to the oro-facial-digital syndrome

TBC1D32基因中的两个新突变增加了口面指综合征的复杂性。

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Abstract

BACKGROUND: Ciliopathies are characterized by the dysfunction of cilia, being inherited retinal dystrophies (IRDs) included in sensory ciliopathies. Besides, oro-facial-digital syndrome (OFD) is caused by mutations in ciliary genes, leading to dysmorphic features. Mutations in TBC1D32 were associated to retinal dystrophy and OFD, defining this form as OFD-IX. RESULTS: A clinical exome analysis performed on a patient presenting with OFD-IX and sensorineural hearing loss (SNHL) identified two variants in TBC1D32, one of which affects splicing, with its impact validated using a minigene assay. CONCLUSIONS: These results suggest that SNHL may represent a new clinical feature associated with this gene.

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