Patient's cells used to replicate dire developmental condition
An impermeable shield of endothelial cells that protects our brains from toxins and other threats that may lurk in circulating blood, the barrier can also exclude therapeutic drugs and, at times, essential biomolecules required for healthy brain development. A case in point is the rare but severe psychomotor disease Allan-Herndon-Dudley syndrome (AHDS), a congenital condition that affects only males and starves the developing brain of thyroid hormone, resulting in cognitive impairment and atrophied muscles and motor skills. The condition is not only untreatable, but seems to be peculiar to humans, meaning scientists have been unable to study the disease and seek new treatments by modeling it in an animal like the mouse. But now, a team of scientists from the University of Wisconsin-Madison and Cedars-Sinai in Los Angeles have used the cells of AHDS patients to recreate not only the disease, but a mimic of the patient's blood-brain barrier in the laboratory dish using induced ...