Genome editing approaches for in vivo gene therapy

Genome editing approaches such as CRISPR/Cas9 system is routinely used to permanently and precisely disrupt potentially any gene of interest. Yet the efficiency of gene editing is still low. Recently we developed a safe and efficient lipid-nanoparticle (LNP) for the delivery of Cas9 mRNA and sgRNAs that utilize our novel amino-ionizable lipid for high-efficiency therapeutic genome editing in aggressive cancer. We plan to expand this line of research to other types of diseases such as rare genetic diseases and blood cancers.