This $486,291 federal Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) supports the development of novel nucleic acid nanoparticle technology to enhance delivery of the CRISPR/Cas9 gene editing system into hematopoietic stem cells. The goal is to improve the effectiveness and precision of genome editing in hematopoietic stem cells, with the potential to revolutionize treatment of genetic blood disorders like sickle cell disease. The project incorporates an interdisciplinary approach combining nucleic acid nanotechnology, computational design, CRISPR technology, and stem cell biology. The award will also involve graduate and undergraduate students at Rutgers University - Camden and the Coriell Institute to support educational objectives. Successful in vitro demonstration of this nanoparticle-based gene delivery system could significantly advance HSCT-based therapies by enabling precise genetic corrections in hematopoietic stem cells.
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