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Why Gene Therapy Is Important

Why Gene Therapy Is Important

Genes that are flawed and do not work properly can cause disease. Gene therapy is a technique for correcting defective genes responsible for disease development. Researchers may use one of several approaches for correcting faulty genes:

  • A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common.
  • An abnormal gene could be swapped for a normal gene through homologous recombination.
  • The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function.
  • The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.

This schematic illustration shows how a human therapeutic gene is inserted into a deactivated mouse retrovirus.

The retrovirus then attaches to and empties its genetic material into a patient's cell (in the laboratory).

The therapeutic human gene is integrated into the patient's DNA and replaces the "defective" gene, in the treatment for ADA.

Image courtesy of NationalCancer Institute    

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