Gene therapy for inborn errors of immunity: Base editing comes into play.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 37001495.
- Also identified by DOI 10.1016/j.cell.2023.03.001 and PMC identifier 10364572.
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Abstract
CRISPR-Cas9-based base editing allows precise base editing to achieve conversion of adenosine to guanine or cytosine to thymidine. In this issue of Cell, McAuley et al. use adenine base editing to correct a single base-pair mutation causing human CD3δ deficiency, demonstrating superior efficiency of genetic correction with reduced undesired genetic alterations compared with standard CRISPR-Cas9 editing.
Medical subject headings
- CRISPR-Cas Systems
- Gene Editing
- Immune System Diseases