Researchers from the University of Trento have discovered a new CRISPR enzyme called CoCas9, which could potentially expand the use of gene editing for therapeutic applications. This compact enzyme, consisting of just over 1,000 amino acids, was found in a bacterium from the human gut microbiome.

1. Through an extensive analysis of metagenome-assembled genomes, primarily from human microbiomes, the researchers uncovered a vast variety of type II CRISPR-Cas loci.
2. Among these, they identified CoCas9, a highly active and precise nuclease with a reduced molecular size, isolated from an uncultivated Collinsella species.
3. Experiments in human hematopoietic stem/progenitor cells and murine retinas demonstrated the potential of CoCas9 for gene editing applications.
4. In murine retinas, CoCas9 achieved up to 35.6% editing efficiency when targeting the RHO gene, which is mutated in a common form of autosomal dominant retinitis pigmentosa.

1. CoCas9's compact size makes it easier to package with guide RNA within the tight quarters afforded by adeno-associated virus (AAV) vectors, enabling in vivo gene editing for therapeutic applications.
2. Discovering naturally evolved enzymes like CoCas9 offers advantages over modifying existing enzymes, expanding the CRISPR toolbox for developing therapies for genetic diseases.
3. The collaboration between molecular virology and computational metagenomics laboratories at the University of Trento has unlocked a vast natural reserve of CRISPR-Cas9 systems for human genome editing.

The discovery of CoCas9 represents a significant step forward in genomic research and the development of therapies for genetic disorders. Its compact size and high efficiency make it a promising candidate for further investigation and optimization through engineering approaches.

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