CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing.
Nat Commun
; 15(1): 3478, 2024 Apr 24.
Article
em En
| MEDLINE
| ID: mdl-38658578
ABSTRACT
The expansion of the CRISPR-Cas toolbox is highly needed to accelerate the development of therapies for genetic diseases. Here, through the interrogation of a massively expanded repository of metagenome-assembled genomes, mostly from human microbiomes, we uncover a large variety (n = 17,173) of type II CRISPR-Cas loci. Among these we identify CoCas9, a strongly active and high-fidelity nuclease with reduced molecular size (1004 amino acids) isolated from an uncultivated Collinsella species. CoCas9 is efficiently co-delivered with its sgRNA through adeno associated viral (AAV) vectors, obtaining efficient in vivo editing in the mouse retina. With this study we uncover a collection of previously uncharacterized Cas9 nucleases, including CoCas9, which enriches the genome editing toolbox.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Sistemas CRISPR-Cas
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Microbiota
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Edição de Genes
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article