Diving into the gene pool : a user's guide to the new genetic medicine / words, Gail Vines.

  • Vines, Gail.
Date:
1995
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Licence: Attribution 4.0 International (CC BY 4.0)

Credit: Diving into the gene pool : a user's guide to the new genetic medicine / words, Gail Vines. Source: Wellcome Collection.

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    REBUILDING GENES Gene therapy - treating or preventing disease by manipulating an individual's genetic material - is now moving out of the realms of fantasy and into the clinic. The idea is to ferry into a patient's body copies of the desired gene, which will produce the missing protein or the missing genetic switch. Progress is being made using specially disabled viruses and other gene- carriers such as liposomes (fatty particles) to ferry healthy human genes into the relevant cells: say, the lung cells of people with cystic fibrosis, or the muscle cells of boys with Duchenne muscular dystrophy. For instance, these viruses could be loaded into an aerosol inhaler, to deliver the good missing genes straight into the mucus-clogged lungs of a person with cystic fibrosis. As the modified viruses infect the lung cells, they would splice the missing gene into the genetic material of each cell, and so enable the cells to work normally. The trick is to get the desired genes into the right cells without damaging the cells' genes. What's more, researchers need to ensure that the new gene works properly once in position, and continues to function normally for as long as possible. Overcoming these problems remain a considerable technical challenge, and gene therapy is unlikely to become a routine treatment during the next ten years. More controversial is the concept of germ-line gene therapy, in which the sperm, egg or embryo itself is genetically modified to alter a disease causing gene. But most researchers and ethical bodies consider this form of manipulation to be unacceptable. Any modifications, intentional or otherwise, would then be passed down through the generations.