Phenotypic and Genetic Heterogeneity of Adult Patients with Hereditary Spastic Paraplegia from Serbia

Cells. 2022 Sep 8;11(18):2804. doi: 10.3390/cells11182804.

Abstract

Hereditary spastic paraplegia (HSP) is among the most genetically diverse of all monogenic diseases. The aim was to analyze the genetic causes of HSP among adult Serbian patients. The study comprised 74 patients from 65 families clinically diagnosed with HSP during a nine-year prospective period. A panel of thirteen genes was analyzed: L1CAM (SPG1), PLP1 (SPG2), ATL1 (SPG3A), SPAST (SPG4), CYP7B1 (SPG5A), SPG7 (SPG7), KIF5A (SPG10), SPG11 (SPG11), ZYFVE26 (SPG15), REEP1 (SPG31), ATP13A2 (SPG78), DYNC1H1, and BICD2 using a next generation sequencing-based technique. A copy number variation (CNV) test for SPAST, SPG7, and SPG11 was also performed. Twenty-three patients from 19 families (29.2%) had conclusive genetic findings, including 75.0% of families with autosomal dominant and 25.0% with autosomal recessive inheritance, and 15.7% of sporadic cases. Twelve families had mutations in the SPAST gene, usually with a pure HSP phenotype. Three sporadic patients had conclusive findings in the SPG11 gene. Two unrelated patients carried a homozygous pathogenic mutation c.233T>A (p.L78*) in SPG7 that is a founder Roma mutation. One patient had a heterozygous de novo variant in the KIF5A gene, and one had a compound heterozygous mutation in the ZYFVE26 gene. The combined genetic yield of our gene panel and CNV analysis for HSP was around 30%. Our findings broaden the knowledge on the genetic epidemiology of HSP, with implications for molecular diagnostics in this region.

Keywords: complicated HSP; genetic testing; hereditary spastic paraplegia; pure HSP.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • DNA Copy Number Variations / genetics
  • Genetic Heterogeneity
  • Humans
  • Kinesins / genetics
  • Membrane Transport Proteins / genetics
  • Neural Cell Adhesion Molecule L1* / genetics
  • Phenotype
  • Prospective Studies
  • Proteins
  • Serbia
  • Spastic Paraplegia, Hereditary* / genetics
  • Spastin / genetics

Substances

  • KIF5A protein, human
  • Membrane Transport Proteins
  • Neural Cell Adhesion Molecule L1
  • Proteins
  • REEP1 protein, human
  • SPG11 protein, human
  • Spastin
  • Kinesins
  • SPAST protein, human

Supplementary concepts

  • Spastic paraplegia 11, autosomal recessive

Grants and funding

This study was funded in part by the Research Foundation-Flanders (FWO): Research grants #G049217N, #G048220N and #G0A2122N (to A.J.) and the Research Fund of the University of Antwerp (Umbrella grant to A.C.).