Our Labs

Work in our lab focuses on immune regulation of graft-versus-host disease (GVHD), a debilitating and potentially fatal complication of hematopoietic stem cell transplantation (HSCT). HSCT can cure high-risk malignancies and other diseases of the blood and bone marrow, yet success is limited as many patients develop this devastating complication.

We aim to elucidate the biological mechanisms underlying immune tolerance in HSCT and develop approaches to enhance regulatory immune cells for GVHD prevention and treatment. Our goal is to use a bench-to-bedside approach to develop a cellular therapy for GVHD, engineering viable approaches to prevention and cure, and thereby make HSCT a safer way to cure cancer and other blood diseases, giving survivors long, healthy lives.

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Up, down and all around. Immune cells are constantly in motion as they seek to defend the host against pathogens. Dramatic cell shape changes induced by alterations in the underlying actin cytoskeleton provide the structural framework required for cell motility.

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Our lab focuses on understanding the physiologic basis for brain injury in neonates, as well as the development of new methodologies for prediction of neurologic injury. The ultimate goal of these efforts is the development of new strategies for neonatal neuroprotection.

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Treatments and outcomes of bone and joint infection in children.

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We utilize a combination of hypothesis-driven and discovery-based approaches to understand factors that regulate host immune responses to infectious and sterile triggers of severe inflammation. We focus on the cytoplasmic recycling pathway of autophagy, which we found protected macrophages against cytokine-induced cell death and mice against fatal cytokine storm syndrome. However, the relative protective or pathogenic role of autophagy in macrophage survival remains unclear in different contexts. Preliminary findings point towards a critical intersection of these processes with immunometabolism. We are leveraging this experience and developing novel tools to understand the commonalities and peculiarities of cytokine storm syndromes triggered by various etiologies including SARS-CoV-2. The ultimate goal is to develop host-directed therapies for infectious and inflammatory disorders.

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The Pediatric Storage Disorders Lab (PDSL), studies the pathogenesis of the neuronal ceroid lipofuscinoses (NCLs, or Batten disease), and other similar neurodegenerative lysosomal storage disorders.

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David Perlmutter is internationally recognized for his research on the pathobiology of α1-antitrypsin deficiency, a rare disease in which a misfolded protein causes chronic liver failure and hepatocellular carcinoma. His work has led to advances in understanding the basic mechanisms of liver fibrosis and carcinoma and novel therapeutic strategies. Together with collaborators he has discovered a pipeline of drugs that can eliminate misfolded proteins and reverse the liver disease in model organisms. One of these drugs has advanced to phase II/III clinical trials. The class of drugs may also be utilized for other diseases caused by misfolded proteins, including Alzheimer’s disease and other age-dependent degenerative diseases.

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Our lab focuses on the pathogenesis of Klebsiella pneumoniae (Kp) — an opportunistic pathogen that is increasingly becoming multidrug resistant. As a result, resistant Kp is deemed “urgent” by the CDC and a “priority pathogen” by the World Health Organization.

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The Rubenstein lab focuses on the mechanisms by which small molecules may improve the function of mutant proteins that cause disease due to abnormal biogenesis and trafficking. These studies initially focused on epithelial ion channels relevant to Cystic Fibrosis, and have expanded to address fundamental biologic questions related to the regulation of protein biogenesis and trafficking in a number of other diseases and conditions.

Research profile