Publications by authors named "Jack Bozik"
Nucleic Acids Res
December 2024
Article Synopsis
- Researchers developed enhanced single-stranded DNA (esDNA) templates with chemical modifications that significantly improve the efficiency of genome editing when used with Cas9, achieving 2-3 times higher knock-in rates compared to standard ssDNA.
- In specific cell types, such as airway basal stem cells and CD34+ hematopoietic cells, esDNA facilitated correction of target genes (CFTR, HBB, CCR5) in over 50% of cases, indicating strong potential for therapeutic applications.
- However, esDNA wasn't effective in induced pluripotent stem cells due to the lack of the nuclease TREX1, suggesting further research is needed for scalable production of modified ssDNA for gene insertion.
