BiophysicsbioRxivPreprint
Nanobodies as therapies for loss-of-function misfolding diseases.
Atanasio Gómez‐Mulas, Mirco Dindo, Juan Luis Pacheco‐García…·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.
DOI 10.1101/2025.04.12.648492