[Analysis of clinical features and FKTN gene variant in a child with congenital muscular dystrophy]

Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2022 Jul 10;39(7):722-726. doi: 10.3760/cma.j.cn511374-20210207-00119.
[Article in Chinese]

Abstract

Objective: To analyze the clinical features and genetic basis for a child featuring elevated creatine kinase (CK).

Methods: Next-generation sequencing (muscular dystrophy related gene panel) was carried out for the proband. Candidate variants were verified by Sanger sequencing of the child and his parents.

Results: The child was found to harbor compound heterozygous variants of the FKTN gene, including a missense c.536G>C (p.R179T) variant from his father and a non-frameshift c.1299_1301delGTG (p.W434del) variant from his mother. Both variants were predicted to be pathogenic.

Conclusion: The compound heterozygous variants of the FKTN gene probably underlay the disease in this child. Above finding has expanded the mutation spectrum of congenital muscular dystrophy.

MeSH terms

  • Child
  • Family
  • High-Throughput Nucleotide Sequencing
  • Humans
  • Membrane Proteins
  • Muscular Dystrophies* / genetics
  • Mutation

Substances

  • FKTN protein, human
  • Membrane Proteins