ÜDS-2010-Autumn-08
Dec. 26, 2010 • 1 min
Gene replacement therapy is being developed for several genetic diseases. Because many difficulties are inherent in treating most serious genetic diseases, scientists have dreamed of developing actual cures. Today, genetic engineering is bringing these dreams closer to reality. Such therapy could take two main forms. One approach would be to introduce copies of a normal gene into a fertilized egg, using modifications of the technology already used to produce transgenic animals. In some transgenic animals the introduced gene can remain stable from generation to generation, constituting a true “genetic cure.” However, this approach raises such complex ethical problems that it is not being actively pursued at this time. A second strategy – to introduce the normal gene into only some body cells (somatic cell gene therapy) – is receiving increased attention today. The rationale is that, although a particular gene may be present in all cells, it is expressed only in some. Expression of the normal allele in only the cells that require it may be sufficient to give a normal phenotype. Although this approach presents a number of technical obstacles, which must be overcome, gene therapies for a number of genetic diseases are undergoing development or are being tested on patients in clinical trials.