Delivering base editors to the liver and lungs in alpha-1 antitrypsin deficiency

We developed Dual Selective Organ-Targeting Lipid Nanoparticles (Dual SORT LNPs) capable of delivering base editors to multiple organs. Base editor Dual SORT LNPs corrected disease-causing mutations in both the liver and lungs, the primary organs affected in alpha-1 antitrypsin deficiency.