On wired Health peak Last week, Harvard Biochemist and the pioneer of the David Liu gene edition said that this year his laboratory is planning to report on one gene edition strategy, which can treat many unrelated diseases. He calls it-agnostic therapeutic genes.
“It sounds a bit crazy, but in fact there is a very good reason for molecular biology, why it can be possible,” he told the audience in Boston, stopping the details.
Currently, gene edition treatments are being developed for several sporadic and hereditary genetic diseases. One treatment of gene editions, called Casgevy, is approved and available in trade for the treatment of sickle -momatic and related blood disorders called beta thalasemia. At the beginning of this year, KJ Muldoon, a boy born with a often fatal genetic disease, which causes ammonia accumulation in the blood, was saved thanks to non-standard treatment of gene edition-first medical.
These treatment act by directing specific mutations associated with these diseases. But they can be high-priced in development and must be designed for specific patient populations. Sometimes patient populations can be very compact, as in the case of a kj child. Its condition, called CPS1 deficiency, affects only one in 1.3 million live births.
Liu provides a future in which one approach to gene edition can be used in many different diseases, regardless of the organ or tissue that affects or their genetic causes. He says that this type of improved strategy is needed because there are so many sporadic diseases collectively and it would be impractical design for each of them. Global genes, a sporadic organization supporting the disease, estimates that there are at least 10,000 sporadic diseases that affect over 400 million people around the world.
Photo: Vail Fucci
“Genetic disease as a whole is not so rare. In fact, it is many times more widespread than cancer or HIV/AIDS,” said Liu. “We urgently need these ways to directly treat the basic cause of these genetic diseases.”