NIH Announces New Viral Vector for Sickle Cell Disease

October 2, 2019

Researchers used advanced animal model lab tests to determine the new vector is 10 times more efficient at incorporating corrective genes into bone marrow stem cells than traditional vectors.

The National Institutes of Health (NIH) announced that its researchers have developed a new viral vector for use in gene therapy in sickle cell disease, a blood disorder caused by a mutation or misspelling in the beta-globin gene. 

According to an Oct. 2, 2019 press release, researchers used advanced animal model lab tests to determine the new vector is 10 times more efficient at incorporating corrective genes into bone marrow stem cells than traditional vectors. Researchers also reported that the carrying capacity of the new vector is six times higher than average.

“Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease,” said study senior author John Tisdale, MD, chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute, in the press release. “It’s the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high efficiency vectors for treating this devastating disorder.”

NIH researchers have been working on bettering beta-globin vectors for nearly 10 years, according to Tisdale. The new and improved vector came about after comparing its lab tests to reverse-oriented vectors. The researchers found that the new vectors could transfer a higher viral load, showed a longer capacity for longevity, and could be produced in higher amounts compared to conventional vectors, the press release said. 

“Our lab has been working on improving beta-globin vectors for almost a decade and finally decided to try something radically different-and it worked,” Tisdale said. “These findings bring us closer to a curative gene therapy approach for hemoglobin disorders.”

Source: NIH