source: Get Science
Gene therapy shows great promise to be potentially transformational treatments for a number of single-gene disorders, such as Duchenne muscular dystrophy, hemophilia and cystic fibrosis.
Gene therapy uses an engineered vector to deliver a replacement copy of a gene to diseased cells. In the case of single-gene disorders, introducing a working gene can help restore function of a faulty or missing protein.
While it’s a straightforward concept, it can be difficult to execute. Successful vectors must be able to carry the gene, which sometimes can be very large; target the right type of cell for a specific disease; insert the new genetic material into the cell; and “activate” the gene to create the missing protein. Scientists also need to engineer vectors to avoid an immune response that could influence how well the therapy works.
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