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The Ahrens-Nicklas lab combines translational studies in patients with molecular, biochemical, and electrophysiologic studies working with animal models to develop novel therapies for rare inherited pediatric disorders.
Research in the Akizu Lab aims to better understand human brain complexity in health and disease, with the ultimate goal of uncovering therapeutic targets for pediatric neurodegenerative disorders.
The Bhoj Lab discovers new human disease genes, their mechanisms, and potential targeted therapies. A bedside-to-bench-to-bedside translational lab that brings discoveries back to patients, we are focused on three novel genes that lead to pediatric neurologic dysfunction: TBCK, H3F3A/B, and MAP4K4.
The Camire Lab is interested in understanding the components of the blood coagulation system, how they interface with activated cells, and how disturbances in their function lead to bleeding and thrombosis. The lab is also interested in developing novel therapeutic approaches (protein, gene-based, small molecule) to mitigate these events which are major causes of morbidity and mortality worldwide.
Our research on childhood onset neurodegenerative diseases is focused on better understanding the biochemistry and cell biology of proteins deficient in these disorders and developing small molecule or gene therapy-based strategies for therapy. We recently demonstrated that the application of recombinant viral vectors to various models of storage disease reversed CNS deficits and improved life span and continue to develop novel vector systems to improve therapeutic outcomes.
The Gadue Lab studies human pancreatic and hematopoietic development and associated diseases using human pluripotent stem cells. The lab’s research efforts are devoted to directed differentiation and CRISPR-based genome engineering of stem cells to use in the study and development of treatments for diabetes and blood disorders.
Dr. George's clinical and research interests are in the development of novel therapeutics for hemophilia. The George Laboratory studies the molecular basis of coagulation with specific emphasis on the intrinsic tenase enzyme complex.
The Lin Lab studies RNA modifications, or epitranscriptomics, in human diseases including cancer. Post-transcriptional RNA processing and modifications are important mechanisms for gene regulation and functional diversity in eukaryotic cells.
Our lab designs novel genetic, chemistry and engineering tools to dissect immune cell-cell and cell-tissue crosstalk and leverages these crosstalk mechanisms to develop biomaterials, protein, and cell-based precision immunotherapies.
The Margaritis Lab uses basic biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions and conduct translational research for the treatment of coagulation defects.
The Sabatino Lab is focused on the inherited bleeding disorder, hemophilia. Our research interests include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic treatment approaches.
Our lab is interested in the hemostasis system, especially coagulation factor VIII and IX, which are deficient in the bleeding disorders hemophilia A and B. By studying these blood proteins, the lab team aims to improve therapies for children with these diseases including gene therapy.
The lab focuses on translational target discovery for a range of neurodegenerative diseases. We combine technology development of large-scale CRISPR-based perturbation screens with application of such technology together with additional genomic approaches.
Research in the Wang Laboratory focuses on the development of bioinformatics methods to improve the understanding of the genetic basis of human diseases, and the integration of electronic health records and genomic information to facilitate genomic medicine on scale.