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Related Experiment Video

Updated: Mar 29, 2026

High Throughput Characterization of Adult Stem Cells Engineered for Delivery of Therapeutic Factors for Neuroprotective Strategies
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Gene therapy for neurological disorders.

Chi-Jing Choong1, Kousuke Baba1, Hideki Mochizuki1

  • 1a Department of Neurology , Osaka University Graduate School of Medicine , Suita , Japan.

Expert Opinion on Biological Therapy
|December 8, 2015
PubMed
Summary

Gene therapy offers a promising approach for treating neurological disorders by correcting genetic defects. Gene silencing techniques show significant potential for future clinical applications in dominant inherited diseases.

Keywords:
Alzheimer’s diseaseGene therapyHuntington’s diseaseParkinson’s diseaseamyotrophic lateral sclerosisstroke

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Area of Science:

  • Neurology
  • Genetics
  • Molecular Biology

Background:

  • Many nervous system disorders lack effective treatments.
  • Gene therapy offers a molecular-level correction for genetic abnormalities.
  • It involves replacing faulty genes or silencing harmful ones.

Purpose of the Study:

  • To review gene therapy techniques for neurological disorders.
  • To discuss challenges and recent advancements in the field.
  • To highlight gene silencing as a key strategy.

Main Methods:

  • Review of current gene therapy techniques.
  • Analysis of potential problems associated with gene therapy.
  • Examination of gene therapy applications in Alzheimer's, Parkinson's, Huntington's, ALS, and stroke.

Main Results:

  • Gene therapy holds revolutionary potential for neurological disease treatment.
  • Overexpression of therapeutic genes is a key strength.
  • Gene silencing strategies are crucial for dominant inherited diseases.

Conclusions:

  • Gene therapy is poised to transform neurological disorder treatment.
  • Significant challenges remain in its widespread application.
  • Gene silencing via antisense oligonucleotides and RNA interference are major breakthroughs with upcoming clinical trials.