Immune system-in-a-dish offers hope for “bubble boy” disease
For infants with severe combined immunodeficiency (SCID), something as simple as a common cold or ear infection can be fatal. Born with an incomplete immune system, kids who have SCID–also known as “bubble boy” or “bubble baby” disease–can’t fight off even the mildest of germs. They often have to live in sterile, isolated environments to avoid infections and, even then, most patients don’t live past a year or two. This happens because stem cells in SCID patients’ bone marrow have a genetic mutation that prevents them from developing critical immune cells, called T and Natural Killer (NK) cells.
Now, Salk researchers have found a way to, for the first time, convert cells from SCID patients to a stem cell-like state, fix the genetic mutation and prompt the corrected cells to successfully generate NK cells in the laboratory. Read more »