This $325,000 project grant awarded by the National Institute of General Medical Sciences (NIGMS) under the Biomedical Research and Research Training program (CFDA 93.859) will support the development and testing of a new class of programmable, non-viral gene therapy carriers and cargo known as virus-inspired DNA origami (VIDO) vectors and repair templates. The project aims to demonstrate that VIDO vectors can effectively encapsulate and co-deliver CRISPR-Cas9 gene editing agents and repair templates, improving knock-in efficiency compared to unstructured controls. The work will establish VIDO vectors as a new, programmable gene therapy platform with advantages over existing alternatives. The grantee, Parabon Nanolabs, Inc., will leverage their nanoengineering expertise to create the VIDO products and a supporting production platform called EssemblixGT. Successful completion of this one-year project could enable researchers to more rapidly design and deploy gene therapies for rare diseases.
Generated 7/16/24, 9:55 AM