ArticlePLoS biology2024
Engineering of a compact, high-fidelity EbCas12a variant that can be packaged with its crRNA into an all-in-one AAV vector delivery system.
Article in PLoS biology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed.
- Molecular mechanisms and biotechnology applications of CRISPR-Cas12a.Nature reviews. Molecular cell biology · 2026Review
- Precise, specific gene editing via a compact GoCas12m-FokI chimeric nuclease.Nucleic acids research · 2026Article
- Review
- The Promise and Pitfalls of AAV-Mediated Gene Therapy for Duchenne Muscular Dystrophy.Current issues in molecular biology · 2025Review
- Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing.Biomaterials · 2025Review
- Article
- Highly parallel profiling of the activities and specificities of Cas12a variants in human cells.Nature communications · 2025Article
- Chemical engineering of CRISPR-Cas systems for therapeutic application.Nature reviews. Drug discovery · 2025Review
- CRISPR detection of cardiac tumor-associated microRNAs.Molecular biology reports · 2025Review
- Prospects for gene therapy in polycystic kidney disease.Current opinion in nephrology and hypertension · 2025Review
- Next-generation CRISPR technology for genome, epigenome and mitochondrial editing.Transgenic research · 2024Review
- Article
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15 authors.
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Abstract
The CRISPR-associated endonuclease Cas12a has become a powerful genome-editing tool in biomedical research due to its ease of use and low off-targeting. However, the size of Cas12a severely limits clinical applications such as adeno-associated virus (AAV)-based gene therapy. Here, we characterized a novel compact Cas12a ortholog, termed EbCas12a, from the metagenome-assembled genome of a currently unclassified Erysipelotrichia. It has the PAM sequence of 5'-TTTV-3' (V = A, G, C) and the smallest size of approximately 3.47 kb among the Cas12a orthologs reported so far. In addition, enhanced EbCas12a (enEbCas12a) was also designed to have comparable editing efficiency with higher specificity to AsCas12a and LbCas12a in mammalian cells at multiple target sites. Based on the compact enEbCas12a, an all-in-one AAV delivery system with crRNA for Cas12a was developed for both in vitro and in vivo applications. Overall, the novel smallest high-fidelity enEbCas12a, this first case of the all-in-one AAV delivery for Cas12a could greatly boost future gene therapy and scientific research.
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Registered trials
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