Researchers at Washington State University in the US identified a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy.
They 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.
Grant Trobridge, associate professor at Washington State University and his 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.”
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.
“Our goal is to develop a safe and effective therapy for SCID-X patients and their families….” said Trobridge.
He predicted that the therapy could be ready for clinical trials within five years.
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