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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.

Research profile

The PSRP was created with the purpose of introducing research to young investigators and to further their existing interest in research. The program includes seminars, mentored research and clinical shadowing experiences during the eight-week scenario, June through July. Selected participants work alongside experienced researchers. Opportunities range from bench to clinical to community-based research.

Uniting clinicians, scientists and trainees, this conference provides a monthly forum for discussing complex cases and advancing research in pediatric immunology. Hosted by the Center for Pediatric Immunology in collaboration with the Jeffrey Modell Diagnostic and Research Center for Primary Immunodeficiencies at St. Louis Children’s Hospital, each session features a challenging case presentation paired with a research talk, fostering dialogue that bridges clinical practice and cutting-edge science at WashU Medicine.

This four-year ACGME-accredited program provides comprehensive clinical training and formal instruction in both pediatrics and medical genetics. Designed for physicians seeking dual certification and academic careers in genetics and genomics, the program offers an integrated pathway to develop expertise across both specialties.

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.

Research profile

Preparing future leaders in pediatric research, PePSTP bridges the transition from MD/PhD completion through clinical training and mentored research. The program combines exceptional clinical experience with rigorous scientific training, offering individualized support, access to subspecialty fellowships, and strong mentorship. Since its launch, PePSTP has grown steadily and continues to receive significant external support.

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.

Research profile

Dedicated to improving the lives of individuals affected by HIV, Project ARK provides comprehensive care, support services and community outreach. Through clinical programs, education and research initiatives, Project ARK works to ensure access to treatment, promote prevention, and advance understanding of HIV/AIDS in the St. Louis region and beyond.

Helping individuals understand implicit attitudes, Project Implicit offers research-based assessments that reveal unconscious preferences and stereotypes. Through online tests and educational resources, the platform promotes self-awareness and supports efforts to reduce bias in personal and professional settings.