Molecular Genetic Analysis of Ukrainian Families with Congenital Cataracts

Abstract

This study was designed to identify the pathogenic variants in five Ukrainian families with autosomal dominant congenital cataracts. Cataracts can be defined broadly as any opacity of the crystalline lens. Lens development is orchestrated by transcription factors. Disease-causing variants in transcription factors and their developmental target genes, including the lens crystallins, are associated with congenital cataracts and other eye diseases. Whole-exome sequencing identified heterozygous disease-causing variants in five Ukrainian families with autosomal dominant congenital cataracts and cosegregation with cataracts was confirmed using Sanger sequencing. Family 97001 showed a missense variant (c.341T>A: p.L114Q) in HSF4; family 97003 showed a missense variant (c.53A>T: p.N18I) in CRYGA; family 97004 showed a missense variant (c. 82G>A: p.V28M) in GJA3; family 97006 showed a missense variant (c.83C>T: p. P28L) in CRYGC; and family 97008 showed a single-base insertion resulting in a frameshift (c.443_444insA: p. Met148IfsTer51) in PAX6. All five families are associated with congenital cataracts. Overall, we report four novel mutations in HSF4, CRYGA, CRYGC and PAX6, and one previously reported mutation in GJA3 that cause autosomal dominant congenital cataracts.

Document Details

Document Type
Pub Defense Publication
Publication Date
Dec 26, 2022
Source ID
10.3390/children10010051

Entities

People

  • James Hejtmancik
  • Mariia Viswanathan
  • Nadiia Fedorivna Bobrova
  • Tatiana Viktorivna Romanova
  • Xiaodong Jiao

Organizations

  • National Eye Institute

Tags

Readers

  • Molecular and genetic basis of cancer.
  • Neurotrauma and Rehabilitation Medicine.

Technology Areas

  • Biotechnology