This post was contributed by guest blogger Harshana S De Silva Feelixge.
Gene therapy technologies hold great promise for improving or potentially curing human diseases that were previously thought to be incurable. Rapid advances in next generation sequencing technologies have allowed scientists to quickly identify underlying genetic causes of some human conditions, opening up new avenues for therapeutics that treat disease at the molecular level. For instance, if a disease is caused by a mutation in a single gene, it can potentially be treated by correcting the mutation or replacing the gene. A notable example is the treatment of Severe Combined Immune Deficiency disease (SCID-XI), also known as bubble boy syndrome. This disease is caused by mutations in the common cytokine receptor gamma chain (c) and is characterized by a lack of immune cell development and function. To date, gene therapy has been used to treat 10 infants with this disease. To do so, their T-cells were grown in vitro, their mutations corrected, and the T-cells were transferred back into the infants. Almost all patients have achieved persistent immunological reconstitution with a normally functioning T cell repertoire (1).