Gene therapy is a treatment that involves introducing genetic material into a patient’s cells to cure disease. It has the potential to help with a variety of conditions, including genetic disorders, cancers, and infectious diseases. For genetic disorders, gene therapy can fix faulty genes, replace missing ones, deactivate problematic genes, or introduce helpful traits.
The first clinical trial occurred in 1990. This trial successfully corrected a faulty gene in the white blood cells of two children with a life-threatening immune disorder. Fast forward to today, and one of those children is still leading a normal life, which shows the success of this therapy. In 2016, Orchard Therapeutics released a commercial gene therapy for this same life-threatening immune disease.
In recent years, gene therapy has made significant advancements. By the end of 2021, regulatory authorities approved 39, with over 60 awaiting approval. We can expect to see many more becoming commercially available soon.
Genetic disorders successfully treated:
- Duchenne muscular dystrophy
- Hyperalphalipoproteinemia 2
- Amyloidosis, hereditary, transthyretin-related
- Hypercholesterolemia, familial 3
- Leber congenital amaurosis
- Spinal muscular atrophy, type III
There are many advantages to gene therapy.
- It can be a one time treatment that permanently cures a disease.
- It can target the root cause of a disease, rather than just treating the symptoms.
- It can treat diseases that are currently untreatable by other methods.
Despite its benefits, these therapies have risks.
- Viral-based therapies can integrate into the genome anywhere and embed themselves in unfavourable regions. This has the potential to cause cancer and other health problems.
- Viral-based therapies often elicit an immune reaction when delivering the genetic material. For one in two people, this means their bodies reject the therapy.
- The long-term effects are still unknown.
Gene therapy is a promising approach to treating disease, but it is important to weigh the risks and benefits before considering it as a treatment option.
