On wired healthy summit Last week, David Liu, a biochemist and gene editing pioneer at Harvard University, said later this year that his lab plans to report a gene editing strategy that can treat many unrelated diseases. He calls it agile therapy gene editing.
“It sounds a little crazy, but there’s actually a good molecular biology reason,” he told the Boston audience.
Gene editing treatments are currently being developed for several rare and inherited genetic diseases. A gene editing therapy called casgevyapproved and commercially available for the treatment of sickle cell disease and related blood diseases called beta thalassemia. Earlier this year, a baby boy KJ Muldoon, who often suffers from a fatal genetic disease, caused ammonia to accumulate in his blood and was preserved in his blood. Customized gene editing therapy– first.
These treatments work against specific mutations associated with these diseases. But their development can be expensive and must be designed for a specific patient population. Sometimes, these patient populations can be small, just like infant KJs. His condition, known as CPS1 deficiency, affected only 1.3 million live births.
Liu envisions a future in which one gene editing method can be used in a variety of different diseases, regardless of the organ or tissue or genetic cause it affects. He said this simplified strategy is needed because there are many rare diseases in general and it is impractical to design treatments for each disease. Global Gene is a rare disease advocacy organization with an estimated at least 10,000 rare diseases affecting more than 400 million people worldwide.
Photo: Vail Fucci
“Overall, it’s not that rare. It’s actually much more than cancer or HIV/AIDS,” Liu said. “We urgently need these ways to treat the root causes of these genetic diseases directly.”
