Lentivirus-mediated Gene Therapy Corrects Ribosomal Biogenesis and Shows Promise for Diamond Blackfan Anemia
Yari Gimenez,Manuel Palacios,Rebeca Sanchez-Dominguez,Christiane Zorbas,Jorge Peral,Alexander Puzik,Laura Ugalde,Omaira Alberquilla,Mariela Villanueva,Paula Rio,Eva Galvez,Lydie Da Costa,Marion Strullu,Albert Catala,Anna Ruiz-Llobet,Jose Carlos Segovia,Brigitte Strahm,Charlotte M. Niemeyer,Cristina Belendez,Thierry Leblanc,Denis L. J. Lafontaine,Juan Bueren,Susana Navarro JCI INSIGHT（2024）
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