CRISPR-Cas9 base editors and their current role in human therapeutics

Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification. This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes. CRISPR-Cas9 BEs are a useful system because of their...

Full description

Saved in:
Bibliographic Details
Published in:Cytotherapy (Oxford, England) England), 2023-03, Vol.25 (3), p.270-276
Main Authors: Lahr, Walker S., Sipe, Christopher J., Skeate, Joseph G., Webber, Beau R., Moriarity, Branden S.
Format: Article
Language:English
Subjects:
base editor
cancer immunotherapy
CRISPR-Cas Systems - genetics
DNA
Gene Editing
gene therapy
Genetic Therapy
hematopoietic stem cell
Humans
multiplex gene editing
sickle cell
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification. This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes. CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction. As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.
ISSN:1465-3249
1477-2566
1477-2566
DOI:10.1016/j.jcyt.2022.11.013