Characterization of a novel SPG3A deletion in a French-Canadian family

Ann Neurol. 2007 Jun;61(6):599-603. doi: 10.1002/ana.21114.

Abstract

Hereditary spastic paraplegias (HSPs) are characterized by progressive lower limb spasticity and weakness. Mutations in the SPG3A gene, which encodes the large guanosine triphosphatase atlastin, are the second most common cause of autosomal dominant hereditary spastic paraplegia. In a large SPG3A screen of 70 hereditary spastic paraplegia subjects, a novel in-frame deletion, p.del436N, was identified. Characterization of this deletion showed that it affects neither the guanosine triphosphatase activity of atlastin nor interactions between atlastin and spastin. Interestingly, immunoblot analysis of lymphoblasts from affected patients demonstrated a significant reduction in atlastin protein levels, supporting a loss-of-function disease mechanism.

Publication types

  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Child, Preschool
  • DNA Mutational Analysis
  • Electrodiagnosis
  • Family
  • GTP Phosphohydrolases / analysis
  • GTP Phosphohydrolases / genetics*
  • GTP-Binding Proteins
  • Humans
  • Lymphocytes / chemistry
  • Membrane Proteins
  • Middle Aged
  • Paraplegia / diagnosis
  • Paraplegia / genetics*
  • Quebec
  • RNA, Messenger / analysis
  • Sequence Deletion / genetics*
  • Two-Hybrid System Techniques

Substances

  • Membrane Proteins
  • RNA, Messenger
  • ATL1 protein, human
  • GTP Phosphohydrolases
  • GTP-Binding Proteins