Portal:Medicine/Selected article/36, 2007



Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.

In the 1980s, advances in molecular biology had already enabled human genes to be sequenced and cloned. Scientists looking for methods of easily producing proteins — such as insulin, the protein deficient in diabetes mellitus type 1 — investigated introducing human genes to bacterial DNA. The modified bacteria then produce the corresponding protein, which can be harvested and injected in people who cannot produce it naturally.

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