Publisher Correction: Base editors for simultaneous introduction of C-to-T and A-to-G mutations

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated enzyme (Cas) is a naturally occurring genome editing tool adopted from the prokaryotic adaptive immune defense system. Currently, CRISPR/Cas9‐based genome editing has been becoming one of the most promising tools for treating human genetic diseases, including cardiovascular diseases, neuro‐disorders, and cancers. As the quick modification of the CRISPR/Cas9 system, including delivery system, CRISPR/Cas9‐based gene therapy has been extensively studied in preclinic and clinic treatments. CRISPR/Cas genome editing is also a robust tool to create animal genetic models for studying and treating human genetic disorders, particularly diseases associated with point mutations. However, significant challenges also remain before CRISPR/Cas technology can be routinely employed in the clinic for treating different genetic diseases, which include toxicity and immune response of treated cells to CRISPR/Cas component, highly throughput delivery method, and potential off‐target impact. The off‐target effect is one of the major concerns for CRISPR/Cas9 gene therapy, more research should be focused on limiting this impact by designing high specific gRNAs and using high specificity of Cas enzymes. Modifying the CRISPR/Cas9 delivery method not only targets a specific tissue/cell but also potentially limits the off‐target impact.

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CRISPR/Cas gene therapy

Zhang

2020

Journal Cellular Physiology

121

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Streamlined process for effective and strand-selective mitochondrial base editing using mitoBEs

Zhang,

Yi,

Tang

et al. 2024

swwlxb

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Mitochondrial base editing tools hold great promise for the investigation and treatment of mitochondrial diseases. Mitochondrial DNA base editors (mitoBEs) integrate a programmable transcription-activator-like effector (TALE) protein with single-stranded DNA deaminase (TadA8e-V106W, APOBEC1, etc .) and nickase (MutH, Nt.BspD6I(C), etc .) to achieve heightened precision and efficiency in mitochondrial base editing. This innovative mitochondrial base editing tool exhibits a number of advantages, including strand-selectivity for editing, high efficiency, and the capacity to perform diverse types of base editing on the mitochondrial genome by employing various deaminases. In this context, we provide a detailed experimental protocol for mitoBEs to assist others in achieving proficient mitochondrial base editing.

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Publisher Correction: Base editors for simultaneous introduction of C-to-T and A-to-G mutations

Cited by 2 publications

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CRISPR/Cas gene therapy

CRISPR/Cas gene therapy

Streamlined process for effective and strand-selective mitochondrial base editing using mitoBEs

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