Homologous Recombination Method and Alveolar Rhabdomyosarcoma
How is it possible to write 470 words that say nothing and also manage to misspell the disease in focus?
The Sydney Morning Herald reports a “new technology” developed by scientists at the University of Utah that is able to knockout genes in mice that were genetically engineered to have alveolar rhabdomyosarcoma, a deadly childhood cancer that affects the large muscles of the trunk, arms, and legs.
The article left me feeling dissatisfied so for more information, I turned to Dr. Mario R. Capecchi’s page at the Eccles Institute of Human Genetics. I think the “new technology” referred to by the article repeatedly is the homologous recombination method.
This technology employs the exchange of DNA sequences, by homologous recombination, between exogenous, newly added DNA sequences and the cognate chromosomal DNA sequences in embryo-derived mouse stem (ES) cells. This process is referred to as gene-targeting. The ES cells containing the desired targeting event are then used to generate mouse germ line chimeras, capable of transmitting the mutation to their progeny.
The homologous recombination method of gene-targeting takes advantage of the natural process of exchanging DNA material between pairs of chromosomes during meiosis and mitosis. In knockout mice, scientists design new genes that replace the mice’s own genes. It’s then possible to study the mice to determine what happens to them without their native genes.
Genetically engineered mice give us the opportunity to study the pathogenesis of human disease and are appropriate test subjects (much more suitable than humans) for developing new therapies. Eventually, gene therapy via homologous recombination method or other techniques will be available to people suffering from such diseases as alveolar rhabdomyosarcoma.
ETA: For one family’s story of their battle with alveolar rhabdomyosarcoma, see Paigee’s Life.
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POSTED IN: Genetic Engineering
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