Nanobodies as therapies for loss-of-function misfolding diseases.
Atanasio Gómez‐Mulas, Mirco Dindo, Juan Luis Pacheco‐García, Silvia Grottelli, Mario Cano‐Muñoz, Pavla Vaňková, Dmitry S. Loginov, EDUARDO C. SALIDO, Petr Man, Francisco Conejero‐Lara, Barbara Cellini, Angel Luis Pey
This study develops and characterizes six nanobodies as potential therapeutics for PH1 by targeting misfolded AGT variants, showing high affinity and functional enhancement in cellular models.
bioRxiv · Apr 12, 2025 · PreprintBiophysics