Center for Basic & Translational Research

The Center for Basic & Translational Research brings investigators together to participate in collaborative, interdisciplinary research environment to study health outcomes and pursue breakthrough treatments. The Center promotes research that combines basic science with clinical expertise to facilitate next-generation treatments for pediatric lung disease and digestive disorders, among others. Our scientists and clinicians work hand-in-hand to pursue innovative treatment models and research.

Regenerative Medicine

Led by Dr. Christine Finck with an emphasis on regenerative technology, researchers are leading the way in Regenerative Medicine at the Center for Basic & Translational Research. Utilizing a patient’s own cells to reduce rejection responses, Dr. Finck and her researchers are focusing on pediatric and neonatal diseases that arise from congenital defects, preterm birth, accidental injuries, and cancer. The Regenerative Medicine research team, using patient-specific stem cell populations and de-cellularized lung scaffolds, have investigated pediatric lung diseases for their causes and potential treatments. This research has expanded into esophageal defects, the use of synthetic polymers and scaffolding as a therapeutic option, and advanced technology for creating in vitro and in vivo 3D bioprinted scaffolds. Dr. Finck and her team use novel 3D bioprinters to create scaffolds that are designed specifically for the host and are technologically reproducible, exploring the next frontier of regenerative science.

Inflammation Biology

Led by Dr. Jeffrey Hyams with an emphasis on basic, clinical, and translational gastroenterology research, Inflammation Biology researchers at the Center for Basic and Translational Research are nationally-recognized leaders and collaborators in multiple NIH- and privately-funded digestive disease research projects. World-class physicians and researchers lead a range of clinical trials to explore the boundaries of scientific discovery for children living with inflammatory diseases, including Crohn’s disease and ulcerative colitis. Dr. Hyams, an internationally recognized pioneer in the fields of gastroenterology treatment and research, leads a team of researchers to understand the biology of these diseases and the best treatment methods for children and families.

Rare Diseases

Dr. Emily Germain-Lee has an extensive clinical and basic science research program focused on finding new therapies and improving the quality of life for patients with rare bone disorders. A major focus of her clinical and translational research has been Albright hereditary osteodystrophy (AHO), a condition caused by mutations in a gene that is crucial to hormonal regulation and skeletal development. Dr. Germain-Lee has clinically evaluated the largest population of patients with this disorder worldwide. By combining clinical research with basic science investigations, she has been able to gain new insights into the AHO phenotype and disease pathogenesis. One of her major contributions was demonstrating that patients with this condition often have evidence of growth hormone (GH) deficiency due to growth hormone-releasing hormone (GHRH) resistance. This finding changed the standard of care for these patients to include GH testing and was part of two FDA R01 clinical trials examining the outcome and potential benefits of GH treatment in this population.

Dr. Germain-Lee also has a translational research program aimed towards finding new treatments for osteogenesis imperfecta (OI, also referred to as brittle bone disease). Based on clinical features in OI of diminished muscle mass secondary to the immobilization that typically accompanies this disorder, Dr. Germain-Lee went on to show that blocking the activin/myostatin signaling pathway in a mouse model of OI can lead to increases in bone and muscle mass. This raised the possibility that drugs targeting this pathway may be a new therapeutic strategy for patients with OI. In fact, this potential treatment strategy is applicable to patients with loss of bone and muscle due to chronic illness of any etiology, including aging. Given that microgravity mimics immobilization with loss of both bone and muscle, Dr. Germain-Lee’s recent investigations with her colleagues surrounding mice that they sent to the International Space Station revealed that this therapeutic strategy led to improvements in both bone and muscle mass in the setting of microgravity. This has implications for treatments to combat the muscle and bone loss occurring concomitantly not only in people afflicted with disuse atrophy on Earth but also in astronauts in space.

Neonatal Microbiome

Led by Dr. Adam Matson, studies on the microbiome are focused on determining the influence of bacterial populations and/or their products on neonatal outcomes and intestinal health in premature infants. His multidisciplinary team integrates state-of-the-art microbial sequencing technology and translational research approaches to track specific pathogens in the neonatal intensive care unit and to characterize metabolic factors that contribute to necrotizing enterocolitis, a catastrophic intestinal disease of prematurity. This research includes the establishment of a Neonatal Specimen Biorepository, allowing several additional research projects ranging from the influence of gut microbes on neurodevelopment to their impact on liver function. In a complementary fashion, Connecticut Children’s Human Milk Research Center is working to better understand the origin of bacterial populations in milk and how they influence establishment of the infant gut microbiota.

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