Recent advancement in genome editing is a promising therapeutic opportunity in cystic fibrosis (CF). Even though potential of CRISPR-Cas strategies has been demonstrated in CF models through correction of CF mutations, the in vivo translation is limited by efficient and safe delivery particularly for targeting the lungs—the primary site of morbidity and mortality in people with CF (pwCF).
We developed cell derived genome editing vesicles (GEV) for CRISPR-Cas delivery. GEV were obtained by engineering extracellular vesicles with membrane anchoring domains and viral envelopes to maximize the cargo capacity and cellular targeting. Using GEVs, we achieved efficient editing of a specific CF mutation in epithelial cell lines, supporting further advancement toward clinical development.

Laboratory of Molecular Virology