Do we have a cure for tuberous sclerosis complex?
The recent development of several mouse models for tuberous sclerosis complex (TSC) provides in vivo systems to test new therapies for the neurological manifestations of TSC. Rapamycin is known to antagonize the effects of loss of TSC protein function in vitro and in mouse TSC models, rapamycin can prevent seizures and improve learning task performance. These findings provide new hope for TSC patients suffering from intractable seizures and possibly, for those with autism and cognitive disabilities.
Crino
Peter B
PB
Department of Neurology, PENN Epilepsy Center, University of Pennsylvania Medical Center Philadelphia, Pennsylvania, USA. peter.crino@uphs.upenn.edu