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Postnatal gene transfer into the central nervous system

J A Wolff1

  • 1Department of Pediatrics, Waisman Center, University of Wisconsin, Madison 53705.

Current Opinion in Neurobiology
|October 1, 1993
PubMed
Summary

Researchers have advanced foreign gene expression in animal central nervous systems using various cell types as vehicles. Direct gene transfer methods, including viral vectors, show promising results for central nervous system gene therapy.

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Area of Science:

  • Neuroscience
  • Molecular Biology
  • Gene Therapy

Background:

  • Foreign gene expression in the central nervous system (CNS) is crucial for understanding neurological diseases.
  • Developing effective gene delivery methods to the CNS remains a significant challenge.

Purpose of the Study:

  • To review progress in foreign gene expression techniques within the CNS of postnatal animals.
  • To highlight the utility of various cell types and direct gene transfer strategies for CNS gene delivery.

Main Methods:

  • Utilizing fetal and adult brain cells as vehicles for gene expression.
  • Employing non-neuronal cells, such as fibroblasts and muscle cells, for CNS gene delivery.
  • Investigating direct gene transfer strategies using viral vectors like herpes and adenoviruses.

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Main Results:

  • Successful expression of foreign genes in the CNS using diverse cellular vehicles.
  • Demonstration of the feasibility of using fibroblasts and muscle cells for CNS gene transfer.
  • Ongoing fruitful investigations into herpes and adenoviral vectors for direct CNS gene delivery.

Conclusions:

  • Significant advancements have been made in expressing foreign genes within the animal CNS.
  • Cellular vehicles and direct gene transfer offer viable approaches for CNS-targeted gene expression.
  • Viral vector-based strategies are a key area of current research for CNS gene therapy.