Gene therapy

  title={Gene therapy},
  author={Richard G. Vile},
  journal={Current Biology},
In its purest form, gene therapy is the delivery and expression of a correct copy of a gene into cells in which the endogenous gene is nonfunctional. It can lead to cure — or significant enhancement of the quality of life — of the patient. The concept is highly attractive as it provides a therapeutic link between laboratory-based studies on the genetic basis of disease and attempts to alleviate patient suffering. In this context, a prototypic disease for gene therapy is cystic fibrosis (CF… CONTINUE READING
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