The University of Arkansas announced Monday that assistant professor of biomedical engineering Christopher Nelson has been awarded more than $1.8 million to study the long-term efficacy of gene therapy.
The five-year award from the National Institutes of Health will be used to consider the genetic and cellular determinants that may be limiting the success of new gene therapies, UA said in a news release.
The goal of the project is to improve the design of gene therapy approaches, with the aim of life-long correction across a range of genetic diseases.
A news release from the university noted that gene-editing tools, like CRISPR, and viruses engineered to safely shuttle replacement human genes into cells are helping doctors make advances in addressing the molecular basis of diseases. The development of these tools is illustrated by ongoing progress in the treatment of diseases like spinal muscular atrophy, inherited blindness and sickle cell disease.
The news release also said there are still many unknowns, including how long gene replacement therapies last, whether the body will fight a therapy with a strong immune response, which can 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 in the release. “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.”
Nelson’s previous work has shown that in the absence of an immune response, CRISPR-mediated genome editing effects only last for the lifetime of an animal. However, an adaptive immune response can reverse this gene therapy. Additionally, his team has observed high levels of the gene-edited material making deeper, unwanted changes to a patient’s genome.
With the grant, Nelson hopes to better understand the long-term consequences of these unwanted changes and how to limit the side-effects, how the different delivery systems for genetic therapies impact the host’s response to them and how different cells and tissues respond to the introduction of gene-edited material.
“If the scientific community can answer these questions, we can build more effective therapies for some of the most devastating genetic diseases,” Nelson said in the release.
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