How Does Gene Therapy Work?


Quick Answer

Gene therapy is an experimental technique performed by replacing a mutated gene with a healthy copy of the gene as an alternative to surgery or medications. It is designed to make a beneficial protein or introduce new genes to mutated cells to compensate for a patient's abnormal genes, explains Genetics Home Reference.

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Full Answer

In some cases, necessary proteins can be missing or defective because of a mutated gene, according to Genetics Home Reference. The protein's natural and healthy state can be restored by substituting the mutated gene with a normal copy of the gene. A doctor cannot simply insert a gene into the cell directly, however. A vector, or carrier, is genetically engineered to deliver and "infect" the cell, thus creating a natural pathway for the normal gene to penetrate and eventually restore the damaged cell.

In a laboratory setting, a sample of the patient's cells is introduced to the vector, notes Genetics Home Reference. The cells holding the vector are returned to the patient, and the new gene makes a functioning protein. Vectors can be given intravenously or injected into the patient's tissue. Gene therapy is highly experimental and not considered a practical treatment approach for disease, but researchers are working to find more effective ways to deliver normal genes to targeted cells.

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