Gene therapy for inborn errors of immunity: Base editing comes into play

Harry L Malech; Luigi D Notarangelo

doi:10.1016/j.cell.2023.03.001

Gene therapy for inborn errors of immunity: Base editing comes into play

Cell. 2023 Mar 30;186(7):1302-1304. doi: 10.1016/j.cell.2023.03.001.

Authors

Harry L Malech¹, Luigi D Notarangelo²

Affiliations

¹ Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA. Electronic address: hmalech@niaid.nih.gov.
² Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA. Electronic address: luigi.notarangelo2@nih.gov.

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.

Publication types

Research Support, N.I.H., Intramural
Comment

MeSH terms

Adenine
CRISPR-Cas Systems* / genetics
Gene Editing*
Genetic Diseases, Inborn / genetics
Genetic Diseases, Inborn / therapy
Genetic Therapy
Humans
Immune System Diseases* / genetics
Immune System Diseases* / therapy
Mutation

Substances

Adenine

Grants and funding

ZIA AI001222/ImNIH/Intramural NIH HHS/United States