Abstract
Targeted gene therapy aims at achieving the expression of therapeutic transgenes in specific and restricted cell populations, thus sparing all other cells of the unwanted effects of the gene product. This strategy is particularly appealing for therapy of the central nervous system (CNS), where many different cell types exist, and where the inappropriate expression of a molecule can produce grave consequences. To accomplish the objectives of targeted gene therapy, two different approaches have been developed. The first one consists in creating vectors that will deliver the transgene exclusively to the selected cells, that is manipulating the transductional capacities of the vector, and the second one is based on the transcriptional properties of the transgene, so that it will only be expressed in cells where the appropriate transcriptional machinery is present. Reaching the goals of targeted gene expression will greatly increase the specificity and safety of gene therapy, thus getting us closer to the fulfillment of the expectations generated by this new branch of molecular medicine.
Keywords: promoter, viral vector, adenovirus, gene transfer, retrovirus, nervous system, transduction, gene transcription, gene therapy
Current Gene Therapy
Title: Gene Therapy Targeting in the Central Nervous System
Volume: 3 Issue: 2
Author(s): Jorge A. Benitez and Jose Segovia
Affiliation:
Keywords: promoter, viral vector, adenovirus, gene transfer, retrovirus, nervous system, transduction, gene transcription, gene therapy
Abstract: Targeted gene therapy aims at achieving the expression of therapeutic transgenes in specific and restricted cell populations, thus sparing all other cells of the unwanted effects of the gene product. This strategy is particularly appealing for therapy of the central nervous system (CNS), where many different cell types exist, and where the inappropriate expression of a molecule can produce grave consequences. To accomplish the objectives of targeted gene therapy, two different approaches have been developed. The first one consists in creating vectors that will deliver the transgene exclusively to the selected cells, that is manipulating the transductional capacities of the vector, and the second one is based on the transcriptional properties of the transgene, so that it will only be expressed in cells where the appropriate transcriptional machinery is present. Reaching the goals of targeted gene expression will greatly increase the specificity and safety of gene therapy, thus getting us closer to the fulfillment of the expectations generated by this new branch of molecular medicine.
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Cite this article as:
Benitez A. Jorge and Segovia Jose, Gene Therapy Targeting in the Central Nervous System, Current Gene Therapy 2003; 3 (2) . https://dx.doi.org/10.2174/1566523034578429
DOI https://dx.doi.org/10.2174/1566523034578429 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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