BioengineeringbioRxivPreprint
Lipid-based nanoparticles deliver mRNA to reverse the pathogenesis of lysosomal acid lipase deficiency in a preclinical model
Matthias Zadory, Elliot Lopez-Vince, Hamza Haddouch…·Davide Brambilla
Screening of a mRNA-LNPs library yielded a formulation with outmost potency and mitigated the progression of LAL deficiency in a preclinical model.