‘Gene Therapy in a Box’ Effective, Reports Nature Communications

October 21, 2016

(Medical Xpress) – A table-top device that enables medical staff to genetically manipulate a patient’s blood to deliver potential new therapies for cancer, HIV and other diseases would eliminate the need for multi-million-dollar “clean rooms,” making gene therapy more possible for even the poorest of countries.  The so-called “gene therapy in a box,” developed by scientists at Fred Hutchinson Cancer Research Center, delivered modified blood stem cells that were as good as—or better—than those manufactured in highly regulated clean rooms—and required less than half the staff, according to a study that will be published on Oct. 20 in Nature Communications. The adapted cells also successfully repopulated the blood system when tested in two different animal models, the study noted. It hasn’t been tested in humans.