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Dr. Ackermann studies diabetes (types 1 and 2) and congenital hyperinsulinism using mouse models, cell lines, and primary human tissue. She aims to identify novel pathways regulating beta cell insulin secretion, leading to innovative therapeutic strategies for these disorders. Current studies include in vivo mouse physiology, ex vivo human islet physiology, CRISPR-Cas9 gene editing, epigenetic modification, and single-cell functional genomics.
Dr. Adams is director of the Comprehensive Vascular Anomalies Program, a cutting-edge, multidisciplinary program that seeks breakthrough treatments and cures for children, adolescents, and young adults with rare, life-threatening tumors and malformations. She is also co-director of the Comprehensive Vascular Anomalies Frontier Program.
Dr. Ahrens-Nicklas works to understand why patients with inherited biochemical disorders often suffer severe, untreatable neurologic and cardiac symptoms. She strives to elucidate the link between biochemistry and network excitability, in order to drive new approaches to therapy.
Dr. Anderson’s research interests focus on the molecular and cellular mechanisms that govern the development of the mammalian forebrain. In his research on the development of the cerebral cortex, he is particularly interested in understanding the molecular underpinnings behind the fate determination and axon targeting of subclasses of GABAergic interneurons implicated in the neuropathology of schizophrenia.
Dr. Argon investigates the unfolded protein response (UPR) , an essential signaling network that determines life or death of stressed cells and tissues. The IRE1 sensor of UPR responds to metabolic stress through four distinct activities and he focuses on determining which stress condition induces each activity and how they are integrated to enable the cells to cope with stress.
The development of gene-based strategies for the treatment of bleeding and thrombotic diseases is at the heart of research by Dr. Arruda. Working collaboratively, Dr. Arruda and his colleagues have carried out early-phase clinical studies on adeno-associated viral vectors for the treatment of severe hemophilia B.