Goldberg Laboratory

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The lab studies mechanisms of cerebral cortical circuit function and circuit dysfunction in neurodevelopmental disorders. Investigators in the lab use mouse and human genetics, electrophysiology, pharmacology, imaging, optogenetics, and behavior, in a range of experimental model systems ranging 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. The lab is particularly interested in the function of a prominent subtype of neuron known as the GABAergic inhibitory interneuron and the role of interneuron dysfunction as a cause of disease. The goal of the lab is to develop novel, mechanistically-oriented treatments and cures for epilepsy syndromes and other neurodevelopmental disorders.

Project Highlights

  • Mechanisms of seizures and epilepsy in Dravet syndrome, a severe childhood-onset epilepsy due to mutation of the sodium channel gene SCN1A. The lab uses electrophysiology, pharmacology, optognetics, and two-photon calcium imaging in acute brain slices and in awake behavior experimental animals (Scn1a+/- mice).
  • Use of cell transplantation as a novel treatment for neurodevelopmental disorders. The lab uses interneuron progenitors engineered from mouse embryonic stem (ES) cells and human iPS cells to treat epilepsy and epilepsy-associated circuit abnormalities in preclinical experimental model systems.
  • Understanding the mechanisms of novel genetic epilepsy syndromes of childhood. Investigators in the Goldberg lab recently discovered de novo mutations in the sodium channel gene SCN3A as a novel cause of severe epilepsy of infancy (epileptic encephalopathy) and are using advanced neuronal models to understand the mechanisms of SCN3Aencephalopathy and attempt to develop novel “precision” approaches to pediatric epilepsy.
Leader

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.