NEW YORK (GenomeWeb News) – Scientists at the Georgia Institute of Technology have been awarded a $16.1 million grant from the National Institutes of Health to develop a zinc finger nuclease (ZFN)-based technology that could be used to correct single-gene disorders.
Georgia Tech said today that scientists at its Nanomedicine Center for Nucleoprotein Machines and its partners will use the five-year renewal grant to use ZFNs and DNA correction templates to develop methods that could be used to treat sickle cell disease, a common single-gene disorder.
The researchers plan to engineer ZFNs to cut DNA at a specific site and correction templates within the nuclei of hematopoietic stem cells from the bone marrow of sickle cell patients. They expect to induce a double-strand break in the DNA near the spot of the mutation for sickle cell disease, thereby activating the gene for correction.
If the plan succeeds, when the corrected stem cells are injected back into the body they will produce healthy red blood cells to replace the sickle cells.
"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," Gang Bao, director of the nanomedicine center and a professor at Georgia Tech and Emory University, said in a statement.
"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," Bao added.
"We want to focus on sickle cell disease to demonstrate this approach, but if we are successful, the same approach can be adopted to treat some of the other 6,000 estimated single gene disorders in the world today, such as cystic fibrosis and Tay-Sachs," Bao continued.
In addition to researchers at Georgia Tech and Emory University, collaborators on this project include the Medical College of Georgia, Cold Spring Harbor Laboratory, New York University Medical Center, Massachusetts Institute of Technology, Stanford University, and Harvard University.