We are developing CRISPR tools that can spread through heart muscle for future therapies.
Genome editing therapeutics based on delivering the Cas9-sgRNA complex (RNPs) have the potential to revolutionize medicine and are currently being investigated as treatments for a wide number of genetic diseases. However, a key challenge limiting the development of Cas9 RNP-based therapeutics is their limited diffusion through tissue. Developing RNP delivery vehicles that can solve the tissue diffusion problem is challenging, since new approaches are currently screened in transformed cell culture models which do not accurately reflect the in vivo environment. The goal of this project is to assess the diffusion characteristics and spatial gene editing ability of different Cas9 delivery vehicles in cardiac microphysiological systems, which are anatomically-relevant 3D cell cultures that are more physiologically accurate than traditional cell culture models.
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