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RNA Editing

RNA Editing

RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...

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Updated: Jan 16, 2026

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e

Published on: February 17, 2023

Precision A3G base editors for disease modeling and correction.

Hongzhi Zeng¹, Aidi Liu², Tyler C Daniel³

¹Department of Chemical and Biomolecular Engineering, Rice University, Houston, TX 77005, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy

|January 15, 2026

Summary

This summary is machine-generated.

Engineered APOBEC3G base editors (A3G-BEs) precisely edit cytosine bases in DNA, improving cystic fibrosis modeling and treatment. These enhanced tools overcome limitations of earlier versions, broadening their therapeutic potential.

Keywords:

APOBEC3G CRISPR-Cas9 base editing cystic fibrosis gene editing human bronchial epithelial cells protein engineering reduced bystander editing

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Published on: August 25, 2021

Area of Science:

Molecular Biology
Gene Editing
Biochemistry

Background:

Cytosine base editors (CBEs) facilitate targeted DNA modifications without double-strand breaks.
APOBEC3G base editors (A3G-BEs) show preference for the second cytosine in 5'-CC-3' motifs but face limitations in precision and targeting scope due to bystander editing and SpCas9 PAM constraints.

Purpose of the Study:

To engineer A3G-BE variants with improved precision and expanded targeting capabilities.
To overcome limitations of existing A3G-BEs, including bystander editing and protospacer adjacent motif (PAM) restrictions.

Main Methods:

Linker optimization and rational mutagenesis were employed to engineer A3G-BE variants.
Integration with SpG and SpRY Cas9 effectors relaxed PAM constraints.
Validation involved precise installation and correction of cystic fibrosis mutations in HEK293T cells.

Main Results:

Engineered A3G-BE variants demonstrated enhanced precision for single cytosine editing within CC motifs.
The targeting scope was broadened to previously inaccessible genomic sites.
Precise editing in human bronchial epithelial cells modulated cystic fibrosis transmembrane conductance regulator (CFTR) expression and function.

Conclusions:

Precision A3G-BE variants offer improved tools for gene editing applications.
These engineered editors show promise for modeling and potentially treating genetic diseases like cystic fibrosis.
The enhanced targeting scope expands the utility of base editing in diverse genomic contexts.