In This Section

Ethan M. Goldberg, MD, PhD
Ethan M. Goldberg
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.



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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.

Education and Training

BA, Harvard College (Neurobiology), 1999

PhD, New York University School of Medicine (Physiology and Neuroscience), 2006

MD, New York University School of Medicine, 2008

Titles and Academic Titles

Attending Physician

Assistant Professor of Neurology, Neuroscience, and Pediatrics

Professional Awards

AAN Annual Meeting Resident Research Travel Scholarship, 2011

Samuel Zeritsky Award for Excellence in Research, Department of Neurology, The University of Pennsylvania School of Medicine, 2013

Citizens United for Research in Epilepsy (CURE) Taking Flight Award, 2014

Grass Foundation - AES Young Investigator Travel Award, 2015

Burroughs Wellcome Fund Career Award for Medical Scientists, 2015

American Society for Clinical Investigation Young Physician-Scientist Award, 2017

Publication Highlights

Favero M, Sotuyo NP, Lopez E, Kearney JA, Goldberg EM. A Transient Developmental Window of Fast-Spiking Interneuron Dysfunction in a Mouse Model of Dravet Syndrome. J Neurosci. 2018 Sep; 38(36):7912-7927. PMID: 30104343
Zaman T, Helbig I, Bozović IB, DeBrosse S, Bergqvist AC, Wallis K, Medne L, Maver A, Peterlin B, Helbig KL, Zhang X, Goldberg EM. Mutations in SCN3A cause early infantile epileptic encephalopathy. Ann Neurol. 2018 Apr; 83(4):703-717. PMID: 29466837
Natan RG, Briguglio JJ, Mwilambwe-Tshilobo L, Jones SI, Aizenberg M, Goldberg EM, Geffen MN. Complementary control of sensory adaptation by two types of cortical interneurons. Elife. 2015 Oct; 4. pii: e09868. PMID: 26460542
Tyson JA, Goldberg EM, Maroof AM, Xu Q, Petros TJ, Anderson SA. Duration of culture and sonic hedgehog signaling differentially specify PV versus SST cortical interneuron fates from embryonic stem cells. Development. 2015 Apr; 142(7):1267-78. PMID: 25804737
Bearden D, Strong A, Ehnot J, DiGiovine M, Dlugos D, Goldberg EM. Targeted treatment of migrating partial seizures of infancy with quinidine. Ann Neurol. 2014 Sep; 76(3):457-61. PMID: 25042079