A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy.
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 open-access journal produced by the Nature Publishing Group. The team is translating their findings into a stem cell gene therapy to target a life-threatening immunodeficiency in newborns called SCID-X1, also known as "Boy in the Bubble Syndrome."
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 SCID-X1 only to see five of them develop leukemia.
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 cancer-causing genes.
"Our goal is to develop a safe and effective therapy for SCID-X patients and their families," said Trobridge. "We've started to translate this in collaboration with other scientists and medical doctors into the clinic."
He predicted that the therapy could be ready for clinical trials within five years.
Source: Scientific Reports via www.labmanager.com
For further laboratory breakthroughs please make sure to attend the 16th Annual Laboratory Management and Medicine Congress — MEDLAB Middle East. Featuring 11 CME-accredited multi-disciplinary conference tracks, including laboratory management, molecular diagnostics and microbiology & immunology, alongside 5 new tracks specialising in updates in clinical diagnostics of cardiology and diabetes.
Check out those popular press releases
- Aprobar unas oposiciones de Gestión Procesal
- Pirámide alimenticia y la dieta mediterránea
- Alimentos que te ayudaran a que no te sientas agotado y desanimado
- H. Jameson has just created a new video about the power of perseverance for weight loss and how it is key for losing 40 pounds
- Anniversary, Iconic Honda Gold Wing- GL1800 launched in India