Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia.
Giménez Y, Palacios M, Sánchez-Domínguez R, Zorbas C, Peral J, Puzik A, Ugalde L, Alberquilla O, Villanueva M, Río P, Gálvez E, Da Costa L, Strullu M, Catala A, Ruiz-Llobet A, Segovia JC, Sevilla J, Strahm B, Niemeyer CM, Beléndez C, Leblanc T, Lafontaine DL, Bueren J, Navarro S.
Giménez Y, et al. Among authors: ugalde l.
JCI Insight. 2024 May 22;9(10):e171650. doi: 10.1172/jci.insight.171650.
JCI Insight. 2024.
PMID: 38775150
Free PMC article.