Open access
Aug 2026
Double-stranded DNA donors and CRISPR-Cas9 for universal correction of mutations causing cystic fibrosis in human airway cells
These results demonstrate that low levels of CFTR integration can be made therapeutically relevant by optimizing the designs of gene editing reagents, an essential step toward in vivo gene therapy for CF.
Vrishti Sinha, P. Ayoub, C. Juett et al.
· Molecular Therapy: Nucleic A... · 0 citations