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Gene therapy is the use of DNA as a pharmaceutical agent to treat disease.
It derives its name from the idea that DNA can be used to supplement or alter genes within an individual’s cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene in order to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a “vector”, which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient’s disease.
Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID. Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy.
Although early clinical failures led many to dismiss gene therapy as over-hyped, clinical successes in 2006-2011 have bolstered new optimism in the promise of gene therapy. These include successful treatment of patients with the retinal disease Leber’s Congenital Amaurosis, X-linked SCID, ADA-SCID, adrenoleukodystrophy, chronic myelogenous leukemia (CLL), and Parkinson’s disease. These recent clinical successes have led to a renewed interest in gene therapy, with several articles in scientific and popular publications calling for continued investment in the field.