URL of this page: http://www.nlm.nih.gov/medlineplus/genesandgenetherapy.html

Genes and Gene Therapy

Summary

Genes are the building blocks of inheritance. Passed from parent to child, they contain instructions for making proteins. If genes don't produce the right proteins or don't produce them correctly, a child can have a genetic disorder.

Gene therapy is an experimental technique that uses genes to treat or prevent disease. The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene. Other approaches include

  • Swapping an abnormal gene for a normal one
  • Repairing an abnormal gene
  • Altering the degree to which a gene is turned on or off

Although there is much hope for gene therapy, it is still experimental.

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