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Gene Therapy

What is gene therapy?


Gene therapy is an experimental technique that uses genes to treat or prevent diseases that have no cure. This promising technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

  • Replacing a mutated gene that causes disease with a healthy copy of the gene.
  • Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
  • Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases including Niemann-Pick, the technique is still under study to make sure that it will be safe and effective.

 

Additional Resources:

What Is Gene Therapy? How Does It Work?  fda.gov  (Español)

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NIH: ghr.nlm.nih.gov/primer/therapy/genetherapy

02/06/2020 jjb