BRAINCAKEGene Therapy
Gene therapy is the treatment of a genetic disorder by inserting a functional allele into a patient's cells, so that the cells can make the protein that the faulty allele fails to produce. It is best suited to disorders caused by a single recessive allele, such as cystic fibrosis or severe combined immunodeficiency (SCID).
In somatic cell gene therapy the healthy allele is inserted into body cells, such as the epithelial cells lining the airways or bone marrow stem cells. Only the treated person is affected and the allele is not passed on to their children. The treated cells are eventually replaced, so the treatment often has to be repeated.
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All 17 lessons in Genetic Engineering and Biotechnology · All OCR A-level Biology topics