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Gene Therapy Definition
Treatment methods, most of which remain experimental, that attempt to manipulate genetic structure or gene encoding. Gene therapy targets either germline (gamete) or somatic cells, using vectors to deliver genes within cells. Germline gene therapy aims to prevent a genetic disorder from passing to new generations, while somatic gene therapy targets genetic disorders that already exist in individuals.
Most often the goal of gene therapy is to replace a defective gene with a healthy, functional gene. The vectors typically used are inactivated viruses into which scientists insert the replacement gene. The virus enters the target cell and delivers the gene.
Applications of gene therapy have not been as successful as researchers have hoped was possible, however, and at present the US Food and Drug Administration (FDA) has not approved any gene therapy methods for use in the United States. The effects of gene therapy appear time-limited, and viral vectors often initiate immune responses. Researchers continue to investigate safe and effective mechanisms to therapeutically manipulate genes with the goal of treating or curing genetic disorders.Page last reviewed: