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Related Experiment Video

Updated: Apr 12, 2026

Intramuscular Injections Along the Motor End Plates: A Minimally Invasive Approach to Shuttle Tracers Directly into Motor Neurons
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Current developments in gene therapy for amyotrophic lateral sclerosis.

Joseph M Scarrott1, Saúl Herranz-Martín, Aziza R Alrafiah

  • 1University of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN) , 385 Glossop Road, Sheffield, S10 2HQ , UK.

Expert Opinion on Biological Therapy
|May 12, 2015
PubMed
Summary

Gene therapy shows promise for Amyotrophic Lateral Sclerosis (ALS) by targeting toxic RNA and proteins. While challenges remain, successful animal model treatments offer hope for future ALS therapies.

Keywords:
adeno-associated virusamyotrophic lateral sclerosisgene therapyself-complementary adeno-associated virus serotype 9

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Area of Science:

  • Neuroscience
  • Genetics
  • Biotechnology

Background:

  • Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease with limited treatment options.
  • Riluzole is the sole approved drug, offering only marginal survival benefits.
  • The complex genetic basis of ALS complicates research and therapeutic development.

Purpose of the Study:

  • To review recent advances in gene therapy for ALS.
  • To highlight successful treatments in animal models.
  • To analyze ongoing gene therapy clinical trials for ALS.

Main Methods:

  • Focus on RNA-mediated and protein-mediated toxic gain-of-function mechanisms.
  • Exploration of gene therapy strategies to reduce toxic molecules.
  • Investigation of methods to protect motor neurons via gene expression modulation.

Main Results:

  • Successful gene therapy approaches have been demonstrated in animal models of ALS.
  • Several promising gene therapy technologies are under development.
  • Clinical trials for ALS gene therapy are underway, though patient-ready therapies are still pending.

Conclusions:

  • Significant progress in identifying ALS targets and developing gene therapy technologies.
  • Overcoming technical, biological, economic, and clinical challenges is crucial for patient therapies.
  • Positive outcomes in preclinical studies provide optimism for future ALS gene therapy.