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NIH renews Nanomedicine Center focused on treating single-gene disorders for $16.1 million

The Georgia Tech-led Nanomedicine Center for Nucleoprotein Machines has received an award of $16.1 million for five years as part of its renewal by the National Institutes of Health (NIH). The eight-institution research team plans to pursue development of a clinically viable gene correction technology for single-gene disorders and demonstrate the technology's efficacy with sickle cell disease.

Sickle cell disease is a genetic condition present at birth that affects more than 70,000 Americans. It involves a single altered gene that produces abnormal hemoglobin the protein that carries oxygen in the blood. In sickle cell disease, red blood cells become hard, sticky and "C" shaped. Sickle cells die early, which causes a constant shortage of red blood cells. The abnormal cells also clog the flow in small blood vessels, causing chronic pain and other serious problems such as infections and acute chest syndrome.

"Even though researchers know sickle cell disease is caused by a single A to T mutation in the beta-globin gene, there is no widely available cure," said center director Gang Bao, the Robert A. Milton Chair in Biomedical Engineering in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University. "By directly and precisely fixing the single mutation, we hope to reduce or eliminate the sickle cell population in an individual's blood stream and replace the sickle cells with healthy red blood cells."

The center is one of eight NIH Nanomedicine Development Centers established in 2005 and 2006, a key initiative of the NIH's long-term nanomedicine research goals. The centers have highly multidisciplinary scientific teams that include biologists, physicians, mathematicians, engineers and computer scientists. Through an intense competition, the NIH selected four centers for second phase funding, including the one led by Georgia Tech.

In addition to experts in the Coulter Department of Biom

Contact: Abby Vogel Robinson
Georgia Institute of Technology Research News

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NIH renews Nanomedicine Center focused on treating single-gene disorders for $16.1 million
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