Gene Therapy for Rare Disorders

National Institutes of Health, via Wikimedia Commons.

After decades of promise tempered by setbacks, gene therapies are nowbringing new treatment options for a variety of inherited and acquireddiseases.

Recently, clinical trials at the NIH Clinical Center and St. Jude Children’s Research Hospital have shown that gene therapy can safely correct the immune systems of children born without critical infection-fighting cells. These successes are the result of safety modifications and improvements in gene transfer efficiency and delivery fueled by basic science research. Now, as clinical progress in the field rapidly grows, gene therapies are beginning to benefit those with inherited immunodeficiencies, blood disorders, blindness, neuromuscular disease, and more.

Read my story at IEEE Pulse: Advances in Gene Therapy Offers Hope for Rare Disorders.