Ethan M. Goldberg

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Ethan M. Goldberg, MD, PhD

Attending Physician

Dr. Goldberg's research program focuses on investigating cerebral cortical circuit function and dysfunction in neurodevelopmental disorders. Using a variety of research techniques, Dr. Goldberg has a specific research interest in the workings of neuron subtype called GABAergic inhibitory interneuron and the role of interneuron dysfunction in disease.

Goldberg Lab

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Bio

Dr. Goldberg's research program delves into the function and dysfunction of the cerebral cortical circuit and the impact on neurodevelopmental disorders. He has a particular interest in a subtype of neuron known as the GABAergic inhibitory interneuron and the role of interneuron dysfunction as a cause of disease.

Among the techniques employed by Dr. Goldberg in his research are mouse and human genetics, electrophysiology, pharmacology, imaging, optogenetics, and behavior. These techniques are used in experimental model systems ranging from heterologous cells in culture, neurons generated from induced pluripotent stem (iPS) cells from human patients, and in ex vivo and in vivo in animal models of human disease.

Dr. Goldberg is currently conducting several studies, all with the aim of pinpointing and developing novel treatments and cures for epilepsy syndromes and other neurodevelopmental disorders.

Specifically, Dr. Goldberg and his lab are investigating the mechanisms of seizures and epilepsy in Dravet syndrome, a severe childhood-onset epilepsy due to mutation of the sodium channel gene SCN1A. For this study he is using electrophysiology, pharmacology, optognetics, and two photon calcium imaging in acute brain slices and in awake behaving experimental animals (Scn1a+/- mice).

He is also exploring the use of cell transplantation as a novel treatment for neurodevelopmental disorders, using interneuron progenitors engineered from mouse embryonic stem cells and human iPS cells to treat epilepsy and epilepsy-associated circuit abnormalities in preclinical experimental model systems.

Finally, Dr. Goldberg is working to understand the mechanisms of novel genetic epilepsy syndromes. He and his team recently discovered de novo mutations in the sodium channel gene SCN3A as a cause of severe epilepsy of infancy (epileptic encephalopathy). They are building upon this finding and using advanced neuronal models to understand the mechanisms of SCN3A encephalopathy, all with an eye toward developing novel precision approaches to pediatric epilepsy.