by Felix Bubeck, Dirk Grimm
A new study characterizes and improves a novel small Cas12a variant before adapting it for in vivo genome editing by delivery via adeno-associated virus (AAV) vectors, showcasing the potential of small CRISPR systems and their compatibility with viral vectors. Adopting Cas12a nucleases for AAV-based gene therapy has been difficult due to their large size. A new study in PLOS Biology characterizes and engineers a new Cas12a nuclease variant that is compatible for in vivo genome editing via an all-in-one AAV delivery system.