Gene Therapy: Therapeutic Mechanisms and Strategies

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Gene therapy is a field of medical science that focuses on the genetic modifications of the cells to achieve a therapeutic effect for the treatment of a disease by repairing defective genetic material. The main objective of gene therapy is to fix a genetic disorder at its source. For example, if mutation in a certain gene occurs in an inherited disease which results in the production of a dysfunctional protein, then, gene therapy can be used to incorporate a copy of this gene that is normal and does not contain the mutation, and thus, produces a functional protein. This strategy is used to tre...

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