Grant Trobridge, an associate professor of pharmaceutical sciences, has altered the way a virus carries a beneficial gene to its target cell. The modified viral vectors reduce the risk of cancer and can be used for many blood diseases.
Trobridge and his team report their development in Scientific Reports, an online
Gene therapy holds potential for treating genetic diseases by replacing defective genes with repaired ones. It has shown promise in clinical trials but has also been set back by difficulties delivering genes, getting them to work for a long time and safety issues. A joint French and English trial, for example, successfully treated 17 out of 20 patients with
Trobridge and his colleagues are using a vector developed from a foamy retrovirus, so named because it appears to foam in certain situations. Unlike other retroviruses, they don’t normally infect humans. They also are less prone to activate nearby genes, including genes that might cause cancer.
Retroviruses are a natural choice for gene therapy because they work by inserting their genes into a host’s genome.
With an eye toward making the vector safer, the Trobridge team altered it to change how it interacts with a target stem cell so it would insert itself into safer parts of the genome. They found that it integrated less often near potential
«Our goal is to develop a safe and effective therapy for
He predicted that the therapy could be ready for clinical trials within five years.
Source: https://news.wsu.edu/2016/11/07/gene-therapy-reduces-cancer-risk/