Gene Therapy

The dysfunction of the immune system can cause immune disorders. The dysfunction factor might be hyper/hypo activity of the immune system, misregulation of any of the components constituting the immune system or a hereditary factor. Overall the immune disorders can be characterized into autoimmune diseases, immunodeficiency and allergies. Apart from Immunotherapy and vaccination, gene therapy also can be considered in treatment of some of these immune disorders.

Gene therapy is the experimental technique developed in 1972. The technique employs genes to treat and prevent diseases like AIDS, cancer, genetic disorders and rare diseases. The principle behind the technique can be in 3 ways:

• Introduction of new gene to a body to fight the disease.
• Replacing the disease caused mutated gene with healthy gene copy(Gene Editing)
• Knocking out the improper functioning gene( Gene Silencing)

Gene therapy is the only therapy which can be effective completely however several regulations, ethical considerations and market investments hinder the success of the therapy. Though the debate on the application of gene therapy still prevails, FDA has shown approval in some of the cases. In August 2017, FDA had given approval for a cancer therapy: CAR-T treatment marketed as Kymriah against leukemia. Another CAR-T treatment, marketed as Yescarta, was approved against non-Hodgkin’s lymphoma in October 2017. Since these cases didn’t exactly show the replacement or repair of gene, they were not truly the first cases of gene therapy.

The first US approval was for Luxturna developed by Spark Therapeutics, which is a 1-time treatment against rare, inherited blindness. Several clinical trials continue to test gene therapy options for genetic conditions, cancer and HIV/AIDS. Some of the diseases where gene therapy was a success was spinal muscular atrophy type 1 (SMA1) and junctional epidermolysis bullosa (JEB). Though several companies have trouble receiving complaints from ethical regulatory boards and also seeking investments for research and market of gene therapy products, the option to consider gene therapy for diseases and conditions which are horrific, deadly with no cure is always open.