Therapeutic Potential of Gene Targeting

It is hoped that gene targeting may eventually become useful in treating some human genetic disorders such as hemophilia and Duchenne muscular dystrophy. Treating human disease by the types of genetic approaches mentioned above is termed "gene therapy." This could, in principle, be achieved by replacing the defective gene with a normal copy of the gene in the affected cells of an individual undergoing treatment. In order to make this potential treatment effective it will be necessary to develop technologies that increase the frequency with which targeting occurs. This is currently the subject of much research.

The development of nuclear transfer technology also has opened up the possible alternative method of using homologous recombination for gene therapy based on cell transfer. Gene targeting would be used to replace the defective genes in selected somatic cells in culture, and their nuclei could then be transferred into stem cells. The stem cells can then be differentiated into the affected cell type (for example, into bone marrow cells for hemophilia) and these cells could then be transplanted to patients. see also Embryonic Stem Cells; Gene; Gene Therapy; Marker Systems.

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