Gene Therapy: Vector Design and Current Challenges
Gene transfer is an approach to the treatment of human diseases based on the delivery and expression of genetic material into somatic cells. Gene delivery can be achieved in vivo through direct introduction into cells or ex vivo through manipulation of cells in culture. In this sense, gene therapy is a new form of drug delivery, in which the drug is recombinant protein synthesized by transduced cells. Gene therapy can be viewed as an extension of conventional medical therapies in which the genetic material is the therapeutic agent. Gene therapy is being applied to many human diseases, and it offers the potential to cure diseases, either as a single treatment modality or as an adjuvant to existing treatments. However, gene therapy is still in its infancy, and many of these promises have not yet been fulfilled.
KeywordsCystic Fibrosis Gene Therapy Gene Delivery Peripheral Artery Disease Nonviral Vector
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