University of Texas Researchers Develop Miniature CRISPR for Enhanced Gene Editing in Human Cells
Researchers at the University of Texas at Austin have engineered a compact version of the CRISPR-Cas12f nuclease, which shows promise for efficient gene editing in human cells. This development addresses a significant challenge in gene therapy: the large size of traditional CRISPR nucleases, which limits their delivery via adeno-associated virus (AAV) vectors. The newly developed Cas12f variant, named Al3Cas12f, is approximately one-third the size of the commonly used Cas9 enzyme. Despite its smaller size, Al3Cas12f demonstrated robust activity, achieving over 50% editing efficiency at numerous genomic sites and exceeding 90% at several targets. The research, published in Nature Structural & Molecular Biology, highlights the potential of this miniature enzyme to overcome current limitations in in vivo gene therapy applications.