Infantile-onset ascending hereditary spastic paralysis is associated with mutations in the alsin gene

Am J Hum Genet. 2002 Sep;71(3):518-27. doi: 10.1086/342359. Epub 2002 Jul 26.

Abstract

We studied 15 patients, from 10 families, who presented with severe spastic paralysis with an infantile onset and an ascending progression. Spastic paraplegia began during the first 2 years of life and extended to upper limbs within the next few years. During the first decade of life, the disease progressed to tetraplegia, anarthria, dysphagia, and slow eye movements. Overall, the disease was compatible with long survival. Signs of lower motor-neuron involvement were never observed, whereas motor-evoked potentials and magnetic resonance imaging demonstrated a primitive, pure degeneration of the upper motor neurons. Genotyping and linkage analyses demonstrated that this infantile-onset ascending hereditary spastic paralysis (IAHSP) is allelic to the condition previously reported as juvenile amyotrophic lateral sclerosis at the ALS2 locus on chromosome 2q33-35 (LOD score 6.66 at recombination fraction 0). We analyzed ALS2, recently found mutated in consanguineous Arabic families presenting either an ALS2 phenotype or juvenile-onset primary lateral sclerosis (JPLS), as a candidate gene. In 4 of the 10 families, we found abnormalities: three deletions and one splice-site mutation. All the mutations lead to a truncated alsin protein. In one case, the mutation affected both the short and the long alsin transcript. In the six remaining families, absence of cDNA ALS2 mutations suggests either mutations in regulatory ALS2 regions or genetic heterogeneity, as already reported in JPLS. Alsin mutations are responsible for a primitive, retrograde degeneration of the upper motor neurons of the pyramidal tracts, leading to a clinical continuum from infantile (IAHSP) to juvenile forms with (ALS2) or without (JPLS) lower motor-neuron involvement. Further analyses will determine whether other hereditary disorders with primitive involvement of the central motor pathways, as pure forms of spastic paraplegia, could be due to alsin dysfunction.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Age of Onset
  • Base Sequence
  • Child
  • Child, Preschool
  • Consanguinity
  • DNA Mutational Analysis
  • Female
  • Guanine Nucleotide Exchange Factors / genetics*
  • Haplotypes / genetics
  • Humans
  • Infant
  • Lod Score
  • Male
  • Mutation / genetics*
  • Paralysis / epidemiology
  • Paralysis / genetics
  • Paralysis / physiopathology
  • Pedigree
  • Polymorphism, Genetic / genetics
  • Spastic Paraplegia, Hereditary / epidemiology
  • Spastic Paraplegia, Hereditary / genetics*
  • Spastic Paraplegia, Hereditary / physiopathology

Substances

  • ALS2 protein, human
  • Guanine Nucleotide Exchange Factors

Associated data

  • OMIM/105400
  • OMIM/118190
  • OMIM/147450
  • OMIM/182600
  • OMIM/182601
  • OMIM/205100
  • OMIM/308840
  • OMIM/312080
  • OMIM/312900
  • OMIM/312920
  • OMIM/602099
  • OMIM/602433
  • OMIM/602783
  • OMIM/604277
  • OMIM/605280
  • OMIM/606352
  • OMIM/606353
  • OMIM/606439
  • OMIM/606640