Neuroscience Center | Research & Innovation

The F.M. Kirby Neurobiology Center is the largest basic neuroscience research enterprise at a U.S. hospital. Our researchers pursue fundamental questions in neurobiology to understand the brain’s circuitry, learn how disturbances can produce neurodevelopmental disorders, and discover novel therapies to treat the root cause of illness and protect the brain from further damage. Their recent scientific contributions include: gene therapy to restore auditory and vestibular function in an Usher syndrome type 1c mouse model, demonstrating that the majority of infants with the severe epilepsy syndrome Ohtahara syndrome have a genetic etiology, and identifying Otx2 as a master regulator of critical periods throughout the cerebral cortex.

The Translational Neuroscience Center seeks to improve the lives of children and adolescents with nervous system disorders by tackling conditions that once seemed resistant to therapy, such as autism spectrum disorder, muscular dystrophy, and brain tumors. The center provides cutting edge tools and facilities to aid discovery. The Repository Core for Neurological Disorders, for example, is a database and biological specimen bank of high-quality clinical data with paired DNA and protein samples from patients. These data support new translational research, clinical trials, and enhanced collaborations among users. The center’s efforts directly impact clinical care, as is the case with the CDKL5 Clinic, which is helping clinicians understand this ultra-rare disorder’s features and trajectory over time.

The Intellectual and Developmental Disabilities Research Center (IDDRC) at Boston Children’s is one of the oldest members of a national, NIH-funded network of centers seeking to prevent and treat intellectual and developmental disabilities through biomedical and behavioral research.

Our neurosurgical team has also pioneered the development of several procedures with superior outcomes that have changed clinical practice. Pial synangiosis stimulates the development of new blood vessels in the brain of patients with moyamoya and is now the mainstay of moyamoya treatment. ETV/CPC is a surgery that combines two procedures, endoscopic third ventriculostomy (ETV) and choroid plexus cauterization procedure (CPC), to treat hydrocephalus in infants and avoid shunt placement.