Looking to the future of gene therapy for hemophilia A and B

被引：3
作者：
Kaczmarek, Radoslaw [1 ]
Herzog, Roland W. [1 ]
机构：
[1] Indiana Univ Sch Med, Herman B Wells Ctr Pediat Res, Indianapolis, IN 46202 USA
来源：
EXPERT REVIEW OF HEMATOLOGY | 2023年 / 16卷 / 11期
关键词：
Factor VIII; factor IX; gene therapy; hemophilia A; hemophilia B; AAV; gene editing; 1ST;
D O I：
10.1080/17474086.2023.2268279
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
引用
收藏
页码：807 / 809
页数：3
相关论文
共 19 条
  • [1] Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery
    Brown, Harrison C.
    Wright, J. Fraser
    Zhou, Shangzhen
    Lytle, Allison M.
    Shields, Jordan E.
    Spencer, H. Trent
    Doering, Christopher B.
    [J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2014, 1 : 14036
  • [2] IL-15 blockade and rapamycin rescue multifactorial loss of factor VIII from AAV- transduced hepatocytes in hemophilia A mice
    Butterfield, John S. S.
    Yamada, Kentaro
    Bertolini, Thais B.
    Syed, Farooq
    Kumar, Sandeep R. P.
    Li, Xin
    Arisa, Sreevani
    Pineros, Annie R.
    Tapia, Alejandro
    Rogers, Christopher A.
    Li, Ning
    Rana, Jyoti
    Biswas, Moanaro
    Terhorst, Cox
    Kaufman, Randal J.
    de Jong, Ype P.
    Herzog, Roland W.
    [J]. MOLECULAR THERAPY, 2022, 30 (12) : 3552 - 3569
  • [3] Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
    George, Lindsey A.
    Monahan, Paul E.
    Eyster, M. Elaine
    Sullivan, Spencer K.
    Ragni, Margaret, V
    Croteau, Stacy E.
    Rasko, John E. J.
    Recht, Michael
    Samelson-Jones, Benjamin J.
    MacDougall, Amy
    Jaworski, Kristen
    Noble, Robert
    Curran, Marla
    Kuranda, Klaudia
    Mingozzi, Federico
    Chang, Tiffany
    Reape, Kathleen Z.
    Anguela, Xavier M.
    High, Katherine A.
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2021, 385 (21) : 1961 - 1973
  • [4] First hemophilia B gene therapy approved: More than two decades in the making
    Herzog, Roland W.
    VandenDriessche, Thierry
    Ozelo, Margareth C.
    [J]. MOLECULAR THERAPY, 2023, 31 (01) : 1 - 2
  • [5] Huang H., 2019, 22 ANN M AM SOC GEN
  • [6] Gene therapy - are we ready now?
    Kaczmarek, Radoslaw
    [J]. HAEMOPHILIA, 2022, 28 : 35 - 43
  • [7] Treatment-induced hemophilic thrombosis?
    Kaczmarek, Radoslaw
    Herzog, Roland W.
    [J]. MOLECULAR THERAPY, 2022, 30 (02) : 505 - 506
  • [8] Eliminating Panglossian thinking in development of AAV therapeutics
    Kaczmarek, Radoslaw
    Pierce, Glenn F.
    Noone, Declan
    O'Mahony, Brian
    Page, David
    Skinner, Mark W.
    [J]. MOLECULAR THERAPY, 2021, 29 (12) : 3325 - 3327
  • [9] Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
    Mahlangu, J.
    Kaczmarek, R.
    von Drygalski, A.
    Shapiro, S.
    Chou, S. -C.
    Ozelo, M. C.
    Kenet, G.
    Peyvandi, F.
    Wang, M.
    Madan, B.
    Key, N. S.
    Laffan, M.
    Dunn, A. L.
    Mason, J.
    Quon, D. V.
    Symington, E.
    Leavitt, A. D.
    Oldenburg, J.
    Chambost, H.
    Reding, M. T.
    Jayaram, K.
    Yu, H.
    Mahajan, R.
    Chavele, K. -M.
    Reddy, D. B.
    Henshaw, J.
    Robinson, T. M.
    Wong, W. Y.
    Pipe, S. W.
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2023, 388 (08) : 694 - 705
  • [10] Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer
    Nathwani, Amit C.
    Reiss, Ulrike
    Tuddenham, Edward
    Chowdary, Pratima
    McIntosh, Jenny
    Riddell, Anne
    Pie, Jun
    Mahlangu, Johnny N.
    Recht, Michael
    Shen, Yu-Min
    Halka, Kathleen G.
    Meagher, Michael M.
    Nienhuis, Arthur W.
    Davidoff, Andrew M.
    Mangles, Sarah
    Morton, Chris L.
    Zhou Junfang
    Radulescu, Vlad Calin
    [J]. BLOOD, 2018, 132
12