Applications of CRISPR-Cas9 for disease research

The ability to sequence and edit DNA is fundamental to understanding the role of genetic elements in normal biology and disease. A microbial adaptive immune system, CRISPR-Cas9, was recently repurposed to enable the precise editing of the mammalian genome. This has not only simplified and expanded genome editing applications but has reduced the cost and speed at which model organisms can be generated. To further the potential of this technology for biomedical research we have established a CRISPR-Cas9 knockin mouse and demonstrated its potential for in vivo gene editing by viral- and non-viral-mediated delivery of guide RNA into neurons, pneumocytes, immune cells and endothelial cells. This opens up new avenues for interrogating genetic elements and modeling disease directly in vivo in potentially any tissue or cell type.