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Gene Therapy

What is gene therapy?

Gene therapy is a medical technique that attempts to manipulate or modify a gene’s expression or to alter the biological properties of living cells. Changing these genetic components of an individual serves as a medical approach to treat or prevent diseases and may be used in place of surgery or medication.

One gene therapy method is gene addition, which introduces a new gene into cells that allows the body to fight diseases. Gene transfer is another method in gene therapy that presents a non-faulty copy of a gene to replace an altered and disease-causing gene.

A newer technique within gene therapy is genome editing, which introduces molecular tools to change existing cell DNA. This may help turn genes on/off or fix genetic alterations that underlie a disorder.

Various gene therapy products include:

  • viral vectors
  • plasmid DNA
  • human gene editing technology
  • bacterial vectors
  • patient-derived cellular gene therapy products

How does gene therapy improve healthcare?

Gene therapy seeks to replace or fix faulty genes causing disease, offering a way to cure a disease with a genetic cause or help the body better fight disease.

Relatively few gene therapies have FDA approval, but the clinical trials indicate there is potential in treating a wide range of conditions, including spinal muscular atrophy, cystic fibrosis, cancer, diabetes, heart disease, AIDS, and hemophilia.