The next gene editing era will be a sick agnostic

On the wired Health summit Last week, Harvard Biochemist and Generating Pioneer David Liu, later this year his laboratory plans to report on a gene editing strategy that could treat many unrelated diseases. He calls it a disease by agnostic decoration of therapeutic genes.

“Sounds kind of crazy, but there’s actually a very good molecular biology, why this could be possible,” said the audience in Boston, stopping the details.

Gene arrays are currently developing for several few and inherited genetic diseases. One treatment for gene editing, called CaskicIt is commercially approved and is available for the treatment of sick disease and a related blood disorder called Beta Thalasemia. Earlier this year, KJ Muldoon, Baby born with often fatal genetic diseases causing ammonia to build in its blood, it has been preserved with Custom treatment for gene editing– And medical first.

These treatments work by targeting certain mutations related to these diseases. But they can be expensive and must be designed for specific patient populations. Sometimes these patient populations can be very small, as in the case of baby KJ. His condition, called the lack of CPS1, affects only one in 1.3 million living births.

Liu is a predicted future in which one approach of gene editing can be used on several different diseases, regardless of which organ or tissue affects or their genetic cause. It says that this type of simplified strategy is needed because it is collective, there are so many few diseases and it would be impractical for treatment for each. Global genes, a rare organization of disease advocacy, assesses that there are at least 10,000 few diseases that affect more than 400 million people around the world.

Photo: Val Fucci

“Genetic disease as a whole is not so rare. In fact, it prevails from cancer or HIV / AIDS,” Liu said. “We urgently need these ways to act directly with the basic cause of these genetic diseases.”