Nature Medicine
Patients have long received cancer treatments at the maximum tolerated dose on a regular schedule. Could a more sophisticated approach save lives?
The most common genetic killer of infants, a disease known as spinal muscular atrophy, is caused by mutations in a single gene. The human genome contains its own backup system — near-identical copies of the defective gene — yet these secondary sequences rarely get used correctly.
More than a decade after Japanese scientists implanted the first bioengineered blood vessel into a child with a congenital heart defect, the experimental treatment has finally made its way into clinical testing in the United States.