Genetic Engineering

HuntingtonÆs disease is an autosomal dominant disease, and until now, efforts at genetic engineering in these patients has been directed at decreasing expression of the abnormal gene (1:24). With the new RNAi technology, an animal model in which small interfering RNA (siRNA) is tailored to a specific gene sequence that hybridizes to the complementary DNA, inhibiting its translation. The siRNA acts by binding a protein complex which targets mRNA to degrade, preventing it from making its aberrant protein and so preventing disease. It is hoped that the process can be applied to human diseases, not only to prevent them in patients with diseases such as HuntingtonÆs which develop in adulthood, but also to treat the disease in patients in whom it has already developed.

Thalassemia, a form of anemia caused by defec

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nticancer treatments that are better tolerated by patients. The first step was to identify the genes that control maytansinoid production and clone them. The structure of these genes was then altered at the gene level. Plans are being made to combine the maytansinoids with antibodies that target tumors so the maytansinoids can located and enter cancer cells and destroy them without harming other cells. Conventional gene therapy to induce new vessel growth in cardiac patients with blocked vessels or poor circulation uses injections of genes that code for vascular endothelial growth factor, a normal protein which induces new vessel growth (4:28). Researchers have now found a way to stimulate a patientÆs own genes to produce more vessels. They have engineered transcription factors which act directly on a patientÆs DNA to stimulate or block protein production. The transcription factors bind to unique DNA sequences near specific medically important genes and turn protein production up or down, as required. This is preferable to traditional gene therapy because the injected genes only code for one protein, but genes often make a whole series of proteins, and turning the gene on allows it to function normally and make all the ne
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Approximate Word count = 1951
Approximate Pages = 8 (250 words per page)