David T. Curiel, MD, PhD, the Distinguished Professor of Radiation Oncology at Washington University School of Medicine in St. Louis, has received a $2.2 million grant from the National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health (NIH) to support his lab’s research into ways to use viruses as vehicles to deliver the CRISPR gene-editing machinery to the correct location inside the body, such as the liver or lungs. Such a delivery tool could lead to new therapeutics for genetic diseases.
The grant is part of $89 million in total funding for NCATS’ Somatic Cell Genome Editing Program. The program is aimed at improving genome-editing technologies to design new therapies that harness CRISPR and other gene-therapy techniques to improve treatments for a variety of genetic diseases. Curiel’s group is seeking to use these tools to improve therapies for diseases such as hemophilia, a blood clotting disorder; and alpha 1 antitrypsin deficiency, in which the body is lacking an important protein, leading to lung and liver damage.
CRISPR has been described as a cut-and-paste tool that allows scientists to alter the genome sequence with great precision. But the risk of off-target effects is high, and scientists are working to find ways to deliver the editing tools to the right place at the right time and with the right genetic cargo. Curiel’s team is investigating how to use adenovirus to deliver CRISPR as well as the gene of interest for a given genetic disorder.