In vivo multigene editing

Using nanoparticles, Cas9, siRNAs, and miRNAs, we can study how many genes work together to promote disease. How do we do this? By delivering multiple siRNAs, miRNAs, or sgRNAs concurrently in vivo. Why is this important? Many diseases are caused by combinations of genes, not a single gene. For example, we co-delivered two RNAs, each targeting a different cancer pathway, to create a 'targeted combination gene therapy' for cancer. We also designed a five gene therapy for heart disease, and reduced inflammation with Cas9 in vivo.

​Designing RNAs and proteins for gene editing

Using molecular biology, we rationally design the genetic drugs we want to deliver. How do we do this? For example, using 'dead' guide RNAs, we can simultaneously turn off gene A and turn on gene B in the same cell using CRISPR-Cas9. Why is this important? Precisely turning certain genes on, and others off, will allow us to study how gene combinations promote disease.