Pipe, Steven W; Leebeek, Frank W G; Recht, Michael; Key, Nigel S; Castaman, Giancarlo; Miesbach, Wolfgang; Lattimore, Susan; Peerlinck, Kathelijne; Van der Valk, Paul; Coppens, Michiel; Kampmann, Peter; Meijer, Karina; O'Connell, Niamh; Pasi, K John; Hart, Daniel P; Kazmi, Rashid; Astermark, Jan; Hermans, Cedric R J R; Klamroth, Robert; Lemons, Richard; Visweshwar, Nathan; von Drygalski, Annette; Young, Guy; Crary, Shelley E; Escobar, Miguel; Gomez, Esteban; Kruse-Jarres, Rebecca; Quon, Doris V; Symington, Emily; Wang, Michael; Wheeler, Allison P; Gut, Robert; Liu, Ying P; Dolmetsch, Ricardo E; Cooper, David L; Li, Yanyan; Goldstein, Brahm; Monahan, Paul E
(Massachussetts Medical Society, 2023-02-23)
Background: Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby ...