Gene therapy in the central nervous system
Part 1. Genetics
1.7. Gene Therapy
Published Online: 15 NOV 2005
Copyright © 2005 John Wiley & Sons, Ltd
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.
- Published Online: 15 NOV 2005
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.
- Parkinson's disease;