RNA-targeting and gene editing therapies for transthyretin amyloidosis - Aimo, Alberto; Castiglione, Vincenzo; Rapezzi, Claudio; Franzini, Maria; Panichella, Giorgia; Vergaro, Giuseppe; Gillmore, Julian; Fontana, Marianna; Passino, Claudio; Emdin, Michele (Nature reviews cardiology, 2022)
Keywords: Amyloid - genetics; Amyloid - therapeutic use; Amyloid Neuropathies, Familial - complications; Amyloid Neuropathies, Familial - genetics; Amyloid Neuropathies, Familial - therapy; Amyloidosis; Cardiomyopathies - genetics; Cardiomyopathies - therapy; Cardiomyopathy; Gene Editing; Gene expression; Genome editing; Humans; Oligonucleotides, Antisense - genetics; Oligonucleotides, Antisense - therapeutic use; Polyneuropathies - complications; Polyneuropathies - drug therapy; Prealbumin - genetics; Prealbumin - therapeutic use; RNA, Small Interfering