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, Samuel Babity, Nastaran Rezaei, Fatma Moawad, Rita Maria Kenaan El Rahbani, Nemanja Vujić, Vincent Quoc‐Huy Trinh, Dagmar Kratky, Simon‐Pierre Gravel, 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.
bioRxiv · Dec 7, 2024 · PreprintBioengineering