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Methods for gene transfer to the central nervous system.

Boris Kantor1, Rachel M Bailey2, Keon Wimberly2

  • 1Department of Pharmacology, Physiology, and Neuroscience, University of South Carolina, Columbia, SC, USA.

Advances in Genetics
|October 15, 2014
PubMed
Summary

This chapter explores viral vectors for central nervous system (CNS) gene transfer, detailing plasmid, retroviral, adeno-associated virus, adenovirus, and herpes simplex virus capabilities for research and clinical use.

Keywords:
AAVAdenovirusCNSGene therapyLentivirusPlasmidPromoterVector

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Area of Science:

  • Neuroscience
  • Molecular Biology
  • Biotechnology

Background:

  • Gene transfer is crucial for central nervous system (CNS) research and therapies.
  • Viral vectors are commonly modified for efficient CNS gene delivery.
  • Understanding vector capabilities is key for successful gene transfer strategies.

Purpose of the Study:

  • To provide a comprehensive overview of gene transfer tools for the CNS.
  • To describe the capabilities of various viral and non-viral vectors for CNS gene delivery.
  • To highlight the role of regulatory elements in controlling transgene expression.

Main Methods:

  • Review of naturally occurring viruses as gene transfer tools.
  • Description of plasmid, retroviral, adeno-associated virus, adenovirus, and herpes simplex virus vectors.
  • Discussion of focal and global CNS gene transfer strategies.
  • Analysis of short- and long-term gene expression.

Main Results:

  • Various vectors offer distinct advantages for CNS gene transfer.
  • Vector choice impacts the scope and duration of gene expression.
  • cis-acting regulatory elements are critical for precise transgene control.

Conclusions:

  • Diverse vector systems are available for CNS gene transfer.
  • Vector selection and regulatory element design are essential for targeted gene delivery.
  • Advancements in vector technology enhance CNS research and therapeutic potential.