NIH awards $1.8 million to UA assistant professor

The National Institutes of Health awarded more than $1.8 million to Christopher Nelson, assistant professor of biomedical engineering at the University of Arkansas, according to a news release.

Nelson will use the five-year award to consider the genetic and cellular determinants that may be limiting the success of new gene therapies. The goal is to enable the improved design of gene therapy approaches with the aim of life-long correction across a range of genetic diseases, the release shows.

Gene-editing tools, like CRISPR, and viruses engineered to shuttle replacement human genes into cells have helped doctors make rapid advances in addressing the molecular basis of disease, Nelson said. Continued progress in treating spinal muscular atrophy, inherited blindness and hemoglobinopathies, such as sickle cell disease, exemplify the rapid development of these life-saving tools.

However, many unknowns remain, such as how long gene replacement therapies last, whether the body will fight a treatment with a strong immune response, which could be fatal, and whether gene therapies will result in unintentional genomic changes, which can trigger undesirable side effects.

“There’s really two things we’re concerned about safety-wise,” Nelson said. “The first is genomic toxicity. Basically, are we doing more harm than good to the cells we are trying to fix? And then the other is, are we triggering really deadly immune responses? And then the thing we want, efficacy-wise, is for the therapy to last a long time.”