Review
Nature Reviews Genetics 7, 261-276 (April 2006) | doi:10.1038/nrg1829
Focus on: Monogenic disorders
Genetic medicines: treatment strategies for hereditary disorders
Timothy P. O'Connor1 & Ronald G. Crystal1 About the authors
Abstract
The treatment of the more than 1,800 known monogenic hereditary disorders will depend on the development of 'genetic medicines' — therapies that use the transfer of DNA and/or RNA to modify gene expression to correct or compensate for an abnormal phenotype. Strategies include the use of somatic stem cells, gene transfer, RNA modification and, in the future, embryonic stem cells. Despite the efficacy of these technologies in treating experimental models of hereditary disorders, applying them successfully in the clinic is a great challenge, which will only be overcome by expending considerable intellectual and economic resources, and by solving societal concerns about modifications of the human genetic repertoire.
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Author affiliations
- Department of Genetic Medicine, Weill Medical College of Cornell University, 515 East 71st Street, S-1000, New York 10021, USA.
Correspondence to: Ronald G. Crystal1 Email: geneticmedicine@med.cornell.edu
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