Identification of a novel frameshift heterozygous deletion in exon 8 of the PAX6 gene in a pedigree with aniridia

Mol Med Rep. 2016 Sep;14(3):2150-4. doi: 10.3892/mmr.2016.5467. Epub 2016 Jul 5.

Abstract

Aniridia is a congenital, panocular abnormality which is characterized by partial or complete absence of iris and various degrees of iris hypoplasia. Mutations in the PAX6 gene are found in ~90% of cases with aniridia. The human PAX6 gene is located at chromosome 11p13 and encodes a transcriptional regulator that has crucial roles in the development of the eyes, central nervous system and pancreatic islets. The present study performed a clinical and genomic analysis of two families containing multiple cases of aniridia. All exons of the PAX6 gene of the probands were sequenced using the Sanger sequencing technique. A heterozygous non‑stop mutation in exon 14 was identified in the first family, which has been previously reported for a different ophthalmological pathology. This mutation causes on‑going translation of the mRNA into the 3'‑untranslated region. In the second family, a novel frameshift heterozygous deletion in exon 8 was identified.

MeSH terms

  • Aniridia / diagnosis*
  • Aniridia / genetics*
  • Base Sequence
  • Consanguinity
  • Exons*
  • Female
  • Frameshift Mutation*
  • Heterozygote*
  • Humans
  • Male
  • PAX6 Transcription Factor / genetics*
  • Pedigree
  • Sequence Analysis, DNA
  • Sequence Deletion*

Substances

  • PAX6 Transcription Factor