Logo image
Back
CRISPR/Cas9-Based Genome Editing: Understanding Differences in DNA Repair Pathways, Profiles, and Outcomes
Journal article   Open access   Peer reviewed

CRISPR/Cas9-Based Genome Editing: Understanding Differences in DNA Repair Pathways, Profiles, and Outcomes

Samuel N. Effah, Shirley C. Barrera, Nahia Urturi Ortiz, Will Dampier, Michael R. Nonnemacher and Brian Wigdahl
International journal of molecular sciences, v 27(13), 5905
30 Jun 2026
DOI: https://doi.org/10.3390/ijms27135905
Featured in Collection :   Research Supported by Drexel Libraries' OA Programs
url
https://doi.org/10.3390/ijms27135905View
Published, Version of Record (VoR) Open Access Discount via Drexel Libraries Read and Publish Program 2026 Open CC BY V4.0

Abstract

Over a decade of advances in Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) and CRISPR-associated protein 9 (Cas9)-based technologies have culminated in the first-ever FDA-approved CRISPR/Cas-based therapy. Aside from this approved therapy for sickle cell anemia, several CRISPR/Cas-based therapies are currently under development or testing for a range of chronic diseases, including viral diseases like human immunodeficiency virus type 1 (HIV-1) infection, genetic diseases like familial hypercholesterolemia, and cancer. The success of these therapies hinges on the effective delivery of CRISPR/Cas9 components to target regions, efficient Cas endonuclease editing, repair profiles generated, and their resulting outcomes. Here, we discuss the factors that influence the generation of CRISPR/Cas9-generated repair edits, the overall profiles, and outcome prediction(s), as well as the analytical tools that have been developed to date. Finally, how this technology has been used towards a functional HIV-1 cure is discussed.

Metrics

1 Record Views

Details

Logo image