Gene Therapy Repairs Ravaged Immune System

Article here

Researches from the University of California in Los Angeles, and the National Human Genome Research Institute (NHGRI) of the National Institute of Health, found the correlations of Gene therapy and immune system dysfunction. According to Dr. Donald and Dr. Fabio, Gene therapy can safely restore immune function in children with severe combined immunodeficiency (SCID).  Children with SCID are not able to produce healthy and adequate lymphocytes compared to normal kids, thus they are more susceptible to infections. Researchers found that one type of SCID arise from a faulty gene that translates into enzyme adenosine deaminase (ADA). Without this enzyme, toxic compounds build up in the body and in response lymphocyte’s production is inhibited.

There were researches about treating this condition with gene therapy by replacing damaged ADA gene in the blood-forming stem cells found in bone marrow. However, they have had trouble developing methods that effectively raises ADA level and leads to lasting improvements in immune function.

In this case, Dr. Donald and Dr. Fabio treated ten SCID patients with ADA deficiency by isolating blood-forming stem cells from their bone marrow, manipulated the cells ’genes correctly and treated with retroviral vectors, which delivered healthy ADA genes. Later on, the corrected cells were infused back into the patient’s blood stream. Four patients were remained with the same therapy mentioned before. The other 6 additional patients, the doctors modified the treatment. They stopped the enzyme-therapy before the procedure and patients received low-dose of chemotherapy. Doctors state that this step proved to be important because it allows them to find the optimal level for enhancing the efficacy of the corrected stem cells by adjusting the chemotherapy dosage. Out of the 6 patients mentioned before, 3 of them received the refined procedure and have had improved health for up to 5 years and have not needed enzyme replacement injections. The other 3 patients did not have lasting improvements from the procedure. This suggests the scientists to improve the therapy.  

I found this article interesting because based on what I read about this disease, children with this condition have to take several painful weekly injections. This research enables the possibility to find a less painful treatment with better results.