Molecular neuroscience stands at the frontier where rapid progress in molecular genetics can be translated into therapies for neurodevelopmental disabilities for which there are currently no treatments.
One of the most common causes of developmental brain disorders worldwide is deficiency of the hormone thyroxine, caused by a deficiency of iodine. Even in developed countries, maternal hypothyroidism appears to be an important cause of developmental disabilities such as learning disabilities and cerebral palsy.
The Institute has a laboratory devoted to the fundamental understanding of how the thyroid affects the expression of genes in the developing brain, and this work is likely to have broad applications to other disorders. Other Kennedy Krieger research programs focus on understanding the causes and treatments for diverse childhood disorders, such as autism, Rett syndrome, holoprosencephaly, spina bifida, adrenoleukodystrophy, cerebral palsy and learning disabilities. Several laboratories at the Institute work on the neurobiologic mechanism for Rett syndrome, a severe genetic intellectual disabilities disorder that predominantly affects girls. Another laboratory program at the Institute is devoted to developing novel ways to express human genes in experimental models, allowing their regulation to be studied and manipulated.
The Institute's program in molecular neuroscience is making fundamental discoveries about how the brain develops so that these discoveries can be translated into therapies for currently incurable diseases.