(Washington Post via MSN) – Therapies that target genetic illnesses at their root are no longer on the horizon. They are here. More are coming. But even as a growing suite of gene therapy tools are changing individual patients’ lives, many are getting stuck in a medical purgatory because they don’t fit the model for turning breakthroughs into accessible treatments.

Donald Kohn, a pediatric bone marrow transplant physician at the University of California at Los Angeles, has successfully rebuilt children’s immune systems with gene therapy in the clinic for over a decade, but it has not yet become a medicine.

“There are several dozen rare diseases in a similar situation, where there is a therapy that looks good in academic clinical trials. But getting to the end zone of an approved drug is very challenging,” Kohn said. (Read More)