The U.S. National Institutes of Health (NIH) has long invested in basic genetics and genomics research, clinical trials, as well as translational medicine and social science studies, to advance our understanding of this widespread illness to help develop effective therapies.

For example:

"NIH celebrates this enormous milestone in treatment for sickle cell disease, the first human genetic disease that was understood at the protein and DNA levels. Researchers have worked hard to find a long-term, durable therapy for sickle cell disease. Research has enabled the use of gene therapy to make genetic changes in the bone marrow of sickle cell patients, leading to normal red blood cell levels. None of this would be possible without federal investments in basic science research."

Eric Green, M.D., Ph.D., Director of the National Human Genome Research Institute

"We have made some exciting research advances over the years and are ready to collect on our scientific investments in sickle cell research. However, we must remember that these advances need to go hand-in-hand with scalable innovations that will ensure equitable access to life-altering care and that we must continue to engage in additional research endeavors that will minimize or eliminate potential risks that might be associated with these therapies."

Gary H. Gibbons, M.D., Director of the National Heart, Lung, and Blood Institute

The sickle cell disease community has historically been underserved and underacknowledged when it comes to rare genetic conditions, so it is heartening to see sickle cell disease at the forefront of gene therapy. It is critical that people with sickle cell disease who are considering gene therapy fully understand the treatment so they can make an informed decision on whether it is appropriate for them. Patients need accessible, understandable and actionable educational materials to help them make such decisions, as well as support from practitioners and the healthcare system to consider these therapies.

Vence L. Bonham, Jr., J.D., Acting Deputy Director of the National Human Genome Research Institute

More:
NIH statement on new FDA-approved gene therapies for sickle cell disease - National Human Genome Research Institute