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Single chain variable fragment antibodies block aggregation and toxicity induced by familial ALS-linked mutant forms

Ghanashyam D Ghadge1, John D Pavlovic, Sujatha P Koduvayur

  • 1Department of Neurology, University of Chicago Medical Center, 5841 S. Maryland Avenue, MC2030, Chicago, IL 60637, USA.

Neurobiology of Disease
|April 24, 2013
PubMed

Insights

Researchers developed human antibody fragments (scFvs) to target toxic mutant SOD1 protein in familial ALS. These scFvs show potential for treating both familial and sporadic ALS by preventing protein aggregation and motor neuron death.

Area of Science:

  • Neuroscience
  • Molecular Biology
  • Immunology

Background:

  • Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease.
  • Familial ALS (FALS) accounts for ~10% of cases, with ~25% caused by mutations in Cu/Zn superoxide dismutase (SOD1).
  • Mutant SOD1 (mtSOD1) toxicity, likely due to aggregation, is implicated in motor neuron death.

Purpose of the Study:

  • To develop novel therapeutic strategies for ALS targeting mtSOD1.
  • To investigate the potential of antibody-based therapies for FALS and sporadic ALS.
  • To generate and characterize human single chain variable fragments (scFvs) against SOD1.

Main Methods:

  • Generation of human scFvs targeting SOD1.
  • Expression of scFvs as intrabodies within a motor neuron cell line.
  • In vitro assessment of scFv efficacy in preventing mtSOD1 aggregation and toxicity.

Main Results:

  • Isolation of human scFvs capable of interfering with mtSOD1 aggregation.
  • Demonstration that these scFvs reduce mtSOD1-induced toxicity in vitro.
  • Evidence of potential therapeutic application for both FALS and sporadic ALS.

Conclusions:

  • Human scFvs targeting SOD1 show promise as a therapeutic approach for ALS.
  • These scFvs may mitigate motor neuron degeneration caused by mtSOD1.
  • The findings support further investigation of scFvs for ALS treatment, including sporadic forms.

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