South Africa’s ‘Bubble Boy’ receives gene therapy in the US – What is Gene Therapy?

You have probably heard of South Africa’s “Bubble Boy” who is receiving gene therapy in the US. If you haven’t, here’s more information on Ayaan Ahmed Isaacs’ story – South Africa’s “Bubble Boy” receives gene therapy in the US

But what exactly is gene therapy?

Primary immunodeficiencies are disorders in which the immune system has not developed normally, making the patient prone to severe, recurrent, unusual or unresponsive infections. Some of these disorders, such as SCID (severe combined immune deficiency) are life threatening and need urgent management with a stem cell transplant. Sometimes, there is not a proper or timeous match for a bone marrow transplant, leaving the patient’s life at risk.

Gene  therapy is a new treatment in which defective genes are replaced by healthy, functioning genes to treat certain disorders. Although it is still largely experimental, gene therapy has been used in some cases of immune deficiencies as described in the case of Ayaan.