Gene therapy

Gene therapy

Can gene therapy treat osteoarthritis?

Gene therapy is an experimental technique to treat or prevent disease. It is designed to introduce genetic material into cells to compensate for abnormal genes by replacing them with a healthy copy of the gene, by knocking out a mutated gene that is functioning improperly, or by introducing a new gene into the body to help fight a disease.

A carrier called a vector is genetically engineered to deliver the gene to the cells. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell but are modified so they do not cause disease. For arthritis, the vector is injected into joints where it is taken up by individual cells. 

A new vector based on the adeno-associated virus (AAV) is currently being tested in a clinical trial to confirm the safety of using AAV to deliver genes to knee joints of patients with OA. Helper‐dependent adenoviral vectors (HDAds) have also shown promise in animal studies. 

Gene therapy is a promising treatment on the horizon for OA and RA but it currently remains in the experimental phase with no treatments approved for use in Canada. More research is still needed to determine which genes to target and the best way to target them, and whether safe and effective therapies can be developed.

Learn more about gene therapy at these links:

This information was reviewed in August 2020 with expert advice from:

Dr. Lauren King, MSc, MBBS, FRCPC
Rheumatologist, Sunnybrook Health Sciences Centre