An SpC editor targeting pre-mRNA splicing for precise CRISPR control and enhanced antitumor efficacy
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Le résumé fourni par la source
The CRISPR/Cas9 system is a powerful genome editing tool that has the potential to be applied to a variety of biomedical applications. Despite the considerable potential of this gene editing technology, there are numerous safety concerns including the possibility of unpredictable off-target effects. The splicing process, which involves the removal of introns from pre-mRNA and the alignment of exons to produce mature transcripts, is a critical step in gene expression in most eukaryotes. In this study, we present a spliceosome-responsive CRISPR/Cas9 (SpC) editor that utilizes the splicing inhibitor pladienolide B (PB) to regulate pre-mRNA splicing and control the expression of the anti-CRISPR protein AcrIIA4, thereby modulating the activity of the Cas9 nuclease. This approach allows for precise regulation of the gene editing process, thereby effectively mitigating off-target effects. The reliability and robustness of the SpC editor were demonstrated through in vitro and in vivo bioluminescence imaging. Furthermore, a dual-target sgRNA was designed to target the diphtheria toxin A gene, resulting in apoptosis induction and growth inhibition of tumor cells across various types of cancer cells. Our results indicate that this SpC editor has the capacity to precisely regulate tumor cell growth, thus providing new insights and significant implications for cancer gene therapy.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- An SpC editor targeting pre-mRNA splicing for precise CRISPR control and enhanced antitumor efficacy
- Date Crossref
- 26/11/2025
- Éditeur
- Oxford University Press (OUP)
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Xidian University pays non établi dans la noticeUniversité ou école supérieure
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Second Affiliated Hospital of Xi'an Jiaotong University Department of Medical Oncology pays non établi dans la noticeÉtablissement de santé
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Xi'an Jiaotong University pays non établi dans la noticeUniversité ou école supérieure
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School of Life Science and Technology Engineering Research Center of Molecular and Neuroimaging pays non établi dans la noticeUniversité ou école supérieure
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School of Basic Medical Sciences Institute of Medical Engineering pays non établi dans la noticeUniversité ou école supérieure
Xidian University, Department of Medical Oncology — Second Affiliated Hospital of Xi'an Jiaotong University et Xi'an Jiaotong University, avec 2 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.