A new RNA-based control switch to trigger production of therapeutic proteins

Researchers at MIT and Harvard University have developed a new way to selectively turn on gene therapies in target cells, including human cells. The devised technology can detect specific messenger RNA sequences in cells, the detection then triggers production of a specific protein from a transgene, or artificial gene. The transgenes can have dangerous effects when expressed in the wrong cells. One way of distinguishing different types of cells is by reading the RNA sequences inside them, which is different from tissue to tissue. By finding a way to produce transgene only after “reading” specific RNA sequences inside cells, the researchers developed a technology that could fine-tune gene therapies in applications ranging from regenerative medicine to cancer treatment. The study was performed in cells grown in a lab dish. The researchers are currently working on delivery strategies that would allow the RNA components of the system to reach target cells in animal models.