Platelet-Targeted Gene Therapy for Hemophilia A with Inhibitor History.
作者信息
Eapen Mary, Malec Lynn M, Armant Myriam A, Johnson Bryon D, Shi Qizhen, Xu Huiqing, Du Lily M, Jerkins James H, Duffy Lisa J, Bushman Frederic D, Lee Carole, Petrichenko Angelina, Hematti Peiman, Brazauskas Ruta, Jobe Shawn M, Hari Parameswaran N, Wilcox David A
机构信息
Medical College of Wisconsin, Milwaukee, WI
Boston Children's Hospital, Boston, MA.
出版信息
N Engl J Med. 2025 Jan 23;392(4):412-414. doi: 10.1056/NEJMc2415164.
Abstract
摘要
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3
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4
Defining the intensity of conditioning regimens: working definitions.
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Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells.
J Biol Chem. 1988 May 5;263(13):6352-62.
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