NanoCas, a smaller version of CRISPR tested with a single AAV, delivers on-target results

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform gene editing in non-liver tissues, including skeletal muscle, using a single adeno-associated virus (AAV) vector. Experiments in non-human primates (NHPs) resulted in editing efficiencies exceeding 30% in muscle tissues.