Gene therapy

What is Gene Therapy? Gene therapy is a type of treatment that involves the manipulation of the genes in the patient’s cells to treat the cause of the disease rather than managing symptoms.

The changes created to the patient’s genome may involve:

  • Replacing a defective or mutated gene.
  • Introducing a gene into the cell.
  • Inactivating or silencing a disease-causing gene.
  • Editing one or more genes within the patient’s genome.


Methods of delivery used in Gene Therapy

  • Plasmid DNA are naked, circular DNA molecules introduced into the patient’s cells. They function like other DNA materials, making proteins to modulate the disease.
  • Vectors using modified viruses or bacteria that carry and insert the therapeutic genes into the patient’s cells. Viruses are used most often.
  • Human gene editing technology, such as CRISPR.
  • Patient-derived cellular gene therapy products, where cells obtained from the patient are genetically modified using a vector, and then reintroduced into the patient’s body.

Applications of Gene Therapy

The potential for the clinical applications of gene therapy is vast, and research is still underway for several therapeutic indications.

The significance of gene therapy lies in its benefits of being efficacious, the goal of using it as a onetime intervention, and the potential to target rare diseases.

Currently, gene therapy is approved for:

  • Genetic disorders, such as Leber Congenital Amaurosis and spinal muscular atrophy.
  • Cancers, such as acute lymphoblastic leukemia, Mantell cell lymphoma, and refractory large B-cell lymphoma.
  • Rare diseases, including TTR amyloidosis.

Source: Center for Biologics Evaluation and Research. (2018, July 25). What is Gene Therapy? U.S. Food and Drug Administration. Retrieved August 24, 2022, from