Future of genetic medicine22nd April 2006
Strategies for genetic medicines (that is, therapies that use the transfer of DNA and/or RNA to modify gene expression to compensate for an abnormal phenotype) include the use of somatic stem cells, gene transfer, RNA modification and, in the future, embryonic stem cells. Of the approximately 25,000 genes that comprise the human genome, mutations in more than 1,800 have already been identified as causing hereditary disorders.
The main biological barriers to all genetic medicines are delivery and maintenance of the new genetic information. Overcoming these hurdles requires an understanding of: the molecular basis of the disorder, its mode of inheritance, the range of mutations and genotype–phenotype relationships that result in the disease phenotype, how the phenotype is modulated by alternative genes, and how, where and when the disease manifests.
Gene transfer of the normal gene to an individual affected by a monogenic disorder is an obvious strategy for genetic medicine. Although many mouse (and larger animal) models of hereditary disorders have been 'cured' with gene transfer, in practice, correcting human hereditary disorders has proved to be difficult. No genetic medicine has been approved for use in the treatment of any hereditary human disorder, but significant intellectual and economic resources are focused on genetic medicines.
The path of development of ground-breaking therapies that we accept as standard today, such as bone marrow transplantation, monoclonal antibodies, in vitro fertilization and organ transplantation were littered by disappointments say O'Connor and Crystal in Nature; similarly, barriers to success in the development of genetic medicines will be overcome, say the authors. They predict that, within 10 to 20 years, doctors of genetic medicine will take their place in the front lines of treating human disease.
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