Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Ozelo, MC; Mahlangu, J; Pasi, KJ; Giermasz, A; Leavitt, AD; Laffan, M; Symington, E; Quon, DV; Wang, JD; Peerlinck, K; Pipe, SW; Madan, B; Key, NS; Pierce, GF; O'Mahony, B; Kaczmarek, R; Henshaw, J; Lawal, A; Jayaram, K; Huang, M; Yang, X; Wong, WY; Kim, B

Ozelo, MC (通讯作者)，Univ Estadual Campinas, Hemoctr UNICAMP, Rua Carlos Chagas 480,Cidade Univ, BR-13083878 Campinas, SP, Brazil.

NEW ENGLAND JOURNAL OF MEDICINE, 2022; 386 (11): 1013

Abstract

BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor......

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