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  4. Triple-Gene Therapy for Stroke: A Proof-of-Concept in Vivo Study in Rats

Triple-Gene Therapy for Stroke: A Proof-of-Concept in Vivo Study in Rats

Frontiers in Pharmacology, 2018 · DOI: 10.3389/fphar.2018.00111 · Published: February 15, 2018

NeurologyGenetics

Simple Explanation

This study investigates a novel approach to stroke treatment using gene therapy. Researchers explored whether introducing genes that promote nerve cell survival and growth could aid recovery after a stroke in rats. The approach involved delivering genes for VEGF, GDNF, and NCAM, either directly or via umbilical cord blood cells. The study examined the impact of these treatments on brain damage and recovery. The results showed that both direct and cell-mediated gene therapy reduced brain damage and improved recovery in rats after stroke, suggesting a potential new treatment avenue.

Study Duration
3 weeks
Participants
35 mature male Wistar rats
Evidence Level
Not specified

Key Findings

  • 1
    Triple gene therapy (VEGF, GDNF, and NCAM) reduced the volume of brain cortex infarct compared to control groups.
  • 2
    Gene therapy decreased the number of Caspase 3 positive cells, indicating reduced apoptosis in the stroke area.
  • 3
    Gene therapy increased the expression of synaptic proteins (PSD95 and synaptophysin), suggesting improved synaptic function recovery.

Research Summary

The study evaluated the therapeutic efficacy of in vivo and ex vivo triple gene therapy (VEGF, GDNF, and NCAM) in a rat model of stroke. Results showed that both in vivo and ex vivo gene therapy reduced infarct volume and attenuated neural cell death, promoting brain cortex remodeling. The findings suggest that intrathecal injection of genetically engineered UCB-MC over-expressing therapeutic molecules might be a novel avenue for future stroke research.

Practical Implications

Novel Therapeutic Strategy

The study suggests a new approach to stroke treatment using gene therapy to promote brain repair and recovery.

Potential for Clinical Translation

The findings support further research into using genetically engineered UCB-MC as a therapeutic strategy for stroke, with potential for clinical application.

Combination Therapy

Combining cell therapy with gene therapy may offer a synergistic effect, enhancing the therapeutic benefits for stroke patients.

Study Limitations

  • 1
    Immunogenicity of UCB-MC
  • 2
    Level of produced recombinant therapeutic molecules
  • 3
    Possible side effects

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