Also known as: CFTR modulators
Researchers developed a lipid nanoparticle gene-editing approach that inserted a full CFTR gene into airway cells and restored 88% to 100% of normal channel function in a cystic fibrosis lab model.
A lipid nanoparticle gene-editing approach inserted a full healthy CFTR gene into human airway cells and restored 88% to 100% of normal CFTR function in lab tests. The nonviral strategy is intended as a mutation-agnostic path for cystic fibrosis.
Three new CRISPR-based therapies show promise for treating heart failure through mitochondrial enhancement, cystic fibrosis via lipid nanoparticle gene insertion, and elevated cholesterol with single-dose gene editing targeting ANGPTL3.
UCLA researchers developed lipid nanoparticle-based gene editing that inserts a complete CFTR gene into human airway cells, restoring up to 100% of normal channel function in cystic fibrosis laboratory models without viral vectors.
