Viral vectors for gene delivery to the central nervous system

Neurobiol Dis. 2012 Nov;48(2):179-88. doi: 10.1016/j.nbd.2011.09.014. Epub 2011 Oct 7.


The potential benefits of gene therapy for neurological diseases such as Parkinson's, Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay in the onset of severe symptoms would be invaluable to patients suffering from these and other diseases. Significant effort has been placed in developing vectors capable of delivering therapeutic genes to the CNS in order to treat neurological disorders. At the forefront of potential vectors, viral systems have evolved to efficiently deliver their genetic material to a cell. The biology of different viruses offers unique solutions to the challenges of gene therapy, such as cell targeting, transgene expression and vector production. It is important to consider the natural biology of a vector when deciding whether it will be the most effective for a specific therapeutic function. In this review, we outline desired features of the ideal vector for gene delivery to the CNS and discuss how well available viral vectors compare to this model. Adeno-associated virus, retrovirus, adenovirus and herpesvirus vectors are covered. Focus is placed on features of the natural biology that have made these viruses effective tools for gene delivery with emphasis on their application in the CNS. Our goal is to provide insight into features of the optimal vector and which viral vectors can provide these features.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Central Nervous System / physiology*
  • Central Nervous System Diseases / therapy*
  • Dependovirus / genetics
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Genetic Vectors*
  • Herpesviridae / genetics
  • Humans
  • RNA, Viral / genetics
  • Retroviridae / genetics
  • Viruses / genetics*


  • RNA, Viral