Complex HBB gene editing outcomes revealed by a fluorescent reporter cell model
Abstract
expression via GFP and blue fluorescent protein (BFP) tagging. Based on fluorescence intensities, this model can discriminate in-frame alleles, frameshift indels, LDs, and LOA, supporting high-throughput genotype-phenotype mapping. By applying HDR-enhancing agents, we further show that the reporter sensitively captures shifts in LOA outcomes that are missed by other bulk assays. This cell model provides a valuable tool for dissecting gene editing outcomes due to different DNA repair pathways and quantitatively linking editing genotypes to erythroid phenotypes and thus can be used to evaluate the safety of CRISPR/Cas9-based therapies.
Fields of Study & Subjects
CRISPR and Genetic Engineering
Virus-based gene therapy research
RNA regulation and disease
Publication details
- Journal / source
- Molecular Therapy — Nucleic Acids
- Publisher
- Elsevier BV
- Volume
- 37
- Issue
- 1
- Pages
- 102854–102854
- Publication date
- 4 February 2026
- Language
- EN