Labs by division

Advancing treatments for blood diseases through innovative cellular therapy research, our center focuses on immune regulation in hematopoietic stem cell transplantation and the development of novel platforms for cell-based therapies. By uncovering mechanisms of immune tolerance, enhancing regulatory immune cells to prevent graft-versus-host disease and designing safer cancer-targeting strategies, we aim to transform HSCT into a more effective and less toxic cure. Our bench-to-bedside approach drives breakthroughs that offer patients long, healthy lives.

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Investigating actin cytoskeleton’s role in immune cell shape and motility our lab studies how dynamic changes in the actin cytoskeleton regulate immune cell movement and activation. Using models lacking the actin-binding protein L-plastin we explore its role in B cells and alveolar macrophages and employ pneumococcal infection models to uncover mechanisms that impact immune defense in children.

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Harnessing data science and artificial intelligence to prevent neonatal brain injury, our lab focuses on understanding neurological damage in newborns and developing predictive tools to guide neuroprotective strategies. By integrating large multimodal datasets — including EHR, NIRS, EEG, MRI and vital signs — we study mechanisms of injury in preterm and term infants and design AI-driven algorithms to improve bedside care. Through clinical trials and collaborative research, we aim to transform outcomes for the most vulnerable patients.

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Advancing care for pediatric bone and joint infections through research on treatments and outcomes, the Orscheln Lab is committed to improving clinical strategies that enhance recovery and long-term health for children. By integrating clinical expertise with innovative research, we aim to identify best practices, optimize therapies and reduce complications associated with musculoskeletal infections.

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Unlocking the mechanisms of immune regulation to combat severe inflammation and cytokine storm syndromes our lab investigates how immune responses are controlled to prevent fatal reactions. We use in vitro and in vivo approaches to identify key genes and pathways, focusing on autophagy and its role in protecting macrophages and preventing cytokine storm. Our goal is to develop host-directed therapies for infectious and inflammatory disorders.

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Leading the way in Batten disease research, the Pediatric Storage Disorders Lab (PSDL) spearheads global efforts to unravel the complexities of neuronal ceroid lipofuscinoses (NCL) and related lysosomal storage disorders. At the forefront of NCL studies and therapeutic innovations, our team combines advanced morphological analysis with cutting-edge approaches such as gene therapy, enzyme replacement and small molecule strategies. By mapping disease progression across diverse models, we aim to refine targeted interventions that offer hope for patients worldwide.

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Transforming the understanding of misfolded protein diseases, David Perlmutter’s laboratory investigates the pathobiology of α1-antitrypsin deficiency, a rare disorder that drives chronic liver failure and hepatocellular carcinoma. Through groundbreaking studies on protein clearance and liver pathology, the team has developed a pipeline of drugs capable of eliminating misfolded proteins and reversing disease in model systems, with one therapy advancing to phase II/III clinical trials. This innovative drug class holds promise not only for liver disorders but also for age-related neurodegenerative diseases such as Alzheimer’s.

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Exploring mechanisms of lung disease and identifying non-CFTR modifiers that influence severity, our lab focuses on airway disorders such as cystic fibrosis to uncover biological pathways driving disease progression. Using molecular biology, genetics, biochemistry and bioinformatics, we investigate factors beyond CFTR that shape inflammatory and immune responses in airway epithelia. Through genomics, multi-omics analyses and translational research, we aim to develop innovative therapeutic targets and personalized strategies to improve outcomes for people with CF and related conditions.

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Fighting multidrug-resistant Klebsiella pneumoniae our lab investigates the pathogenesis of this urgent CDC and WHO priority pathogen and works toward vaccine development. We study classical and hypervirulent strains using mouse models of pneumonia and urinary tract infection to understand virulence factors including capsular types, O-antigens and the fimK regulatory gene. With over 300 clinical isolates in our repository we aim to identify strategies that inhibit virulence or prevent infection through effective vaccines.

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Unveiling ERp29's crucial role in correcting biogenesis defects, our laboratory investigates how small molecules enhance the function of mutant proteins that disrupt normal protein folding and trafficking. Building on discoveries in cystic fibrosis ion channel biology, we now explore broader mechanisms of protein biogenesis regulation across diverse inherited diseases, aiming to identify therapeutic strategies that restore cellular balance.

Research profile