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Gene therapy in the central nervous system

Part 1. Genetics

1.7. Gene Therapy

Specialist Review

  1. Shyam Goverdhana,
  2. Maria G. Castro,
  3. Pedro R. Lowenstein

Published Online: 15 NOV 2005

DOI: 10.1002/047001153X.g107201

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics

How to Cite

Goverdhana, S., Castro, M. G. and Lowenstein, P. R. 2005. Gene therapy in the central nervous system. Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics. 1:1.7:93.

Author Information

  1. University of California, Cedars-Sinai Medical Center, Los Angeles, CA, USA

Publication History

  1. Published Online: 15 NOV 2005

Abstract

Gene therapy for neurological diseases faces many challenges from the anatomy of the brain, that is, its physical encasing by the cranical cavity, and its separation from the bloodstream by the blood brain barrier. This has forced the development of particular ways of delivering genes directly into the brain, to deal with limitations that are not encountered when delivering genes to other organs, such as the liver that can be accessed more easily. Further, the brain is composed of a variety of specialized cell types, such as neurons and glial cells, many of which are postmitotic, this imposing further challenges on the safety standards for gene transfer into the CNS (central nervous system). We here review recent advances in the strategies to develop safe and efficient gene transfer into the CNS for the treatment of degenerative, inherited, and malignant diseases of the brain, and how to make such therapies safe in the presence of potential inflammatory and immune responses.

Keywords:

  • brain;
  • AAV;
  • adenovirus;
  • lentivirus;
  • Parkinson's disease;
  • glioma;
  • inflammation