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Published on: August 28, 2016

Gene therapy for misfolding protein diseases of the central nervous system

Waldy San Sebastian1, Lluis Samaranch, Adrian P Kells

  • 1Department of Neurological Surgery, University of California San Francisco, 1855 Folsom Street, San Francisco, CA, USA.

Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics
|May 24, 2013
PubMed

Insights

Gene therapy offers a promising strategy to combat neurodegenerative diseases by preventing toxic protein misfolding and aggregation. This approach ensures targeted delivery and sustained therapeutic expression for conditions like Alzheimer's and Parkinson's.

Area of Science:

  • Neuroscience
  • Genetics
  • Molecular Biology

Background:

  • Protein misfolding and aggregation are hallmarks of neurodegenerative diseases.
  • Current research focuses on preventing these processes for therapeutic benefit.
  • RNA interference shows promise in preclinical models by reducing protein overexpression.

Purpose of the Study:

  • To review advancements in gene therapy for neurodegenerative diseases.
  • To highlight gene therapy's potential for targeted and sustained treatment delivery in the central nervous system.
  • To discuss recent approaches for treating protein misfolding-related pathologies.

Main Methods:

  • Review of current literature on gene therapy for neurodegenerative diseases.
  • Analysis of state-of-the-art techniques including RNA interference.
  • Evaluation of gene therapy's suitability for central nervous system delivery and chronic expression.

Main Results:

  • Gene therapy demonstrates potential for accurate and long-term therapeutic agent delivery.
  • Successful preclinical outcomes using RNA interference for protein overexpression.
  • Emerging gene therapy strategies are being developed for various protein misfolding diseases.

Conclusions:

  • Gene therapy is a promising approach for treating neurodegenerative diseases.
  • It addresses the need for targeted delivery and chronic expression in the central nervous system.
  • Recent advancements offer new hope for conditions like Alzheimer's, Parkinson's, and Huntington's disease.

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