After three decades of hopes tempered by setbacks, gene therapy—the process of treating a disease by modifying a person’s DNA—is no longer the future of medicine, but is part of the present-day clinical treatment toolkit. The journal Science provides an in-depth and timely review of the key developments that have led to several successful gene therapy treatments for patients with serious medical conditions.

  Scientists at the National Eye Institute (NEI), part of the National Institutes of Health, report that tiny tube-like protrusions called primary cilia on cells of the retinal pigment epithelium (RPE) - a layer of cells in the back of the eye - are essential for the survival of the retina's light-sensing photoreceptors.

In the fight against brain damage caused by stroke, researchers have turned to an unlikely source of inspiration: hibernating ground squirrels. While the animals' brains experience dramatically reduced blood flow during hibernation, just like human patients after a certain type of stroke, the squirrels emerge from their extended naps suffering no ill effects.

Opioid pain relievers can be extremely effective in relieving pain, but can carry a high risk of addiction and ultimately overdose when breathing is suppressed and stops. Scientists have discovered a way to separate these two effects - pain relief and breathing - opening a window of opportunity to make effective pain medications without the risk of respiratory failure. The research, published in Cell, was funded by the Nationa...

Researchers from the NIH Clinical Center Rehabilitation Medicine Department have created the first robotic exoskeleton specifically designed to treat crouch (or flexed-knee) gait in children with cerebral palsy by providing powered knee extension assistance at key points during the walking cycle. Crouch gait, the excessive bending of the knees while walking, is a common and debilitating condition in children with cerebral palsy.

Researchers at the National Institutes of Health's National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a drug originally targeted unsuccessfully to treat cancer may have new life as a potential treatment for Duchenne muscular dystrophy (DMD). The candidate drug, SU9516, represents a different kind of approach for treating DMD, a degenerative muscl...