What is gene therapy?

Gene therapy involves transferring genetic material into a host (human or animal) in an effort to alleviate a disease state. Genetic material is used to modify the expression of a protein(s) essential to either the development or progression of the disease.

Although gene therapy is showing promise for a number of diseases (e.g., inherited disorders, certain cancers and viral infections), the techniques remain experimental and are under study to ensure they are safe and effective. Gene therapy is currently only being tested for treating diseases with no other cures.

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