Optimization of Human FIX Expression Cassettes for Superior Liver-Targeted Gene Therapy of Hemophilia B

被引：0
|
作者：
Liu, Xiaojun [1 ]
Yamada, Kentaro [2 ]
Markusic, David M. [2 ]
Herzog, Roland W. [2 ]
Zhou, Pengcheng [1 ]
Zhang, Jason [1 ]
机构：
[1] Luye Boston Res & Dev LLC, GeneLeap Biotechnol LLC, Woburn, MA USA
[2] Indiana Univ, Dept Pediat, Gene & Cell Therapy Program, Herman B Wells Ctr Pediat Resear, Indianapolis, IN 46204 USA
来源：
MOLECULAR THERAPY | 2021年 / 29卷 / 04期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
340
引用
收藏
页码：168 / 168
页数：1
相关论文
共 50 条
  • [21] Optimization of the human factor VIII complementary DNA expression plasmid for gene therapy of hemophilia A
    Ill, CR
    Yang, CQ
    Bidlingmaier, SM
    Gonzales, JN
    Burns, DS
    Bartholomew, RM
    Scuderi, P
    BLOOD COAGULATION & FIBRINOLYSIS, 1997, 8 : S23 - S30
  • [22] Gene therapy for patients with hemophilia B is superior to factor IX administration
    Franke, Katharina
    DEUTSCHE MEDIZINISCHE WOCHENSCHRIFT, 2023, 148 (23) : 1488 - 1488
  • [23] Oral gene therapy for hemophilia B using chitosan-formulated FIX mutants
    Quade-Lyssy, P.
    Milanov, P.
    Abriss, D.
    Ungerer, C.
    Koenigs, C.
    Seifried, E.
    Schuettrumpf, J.
    JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2014, 12 (06) : 932 - 942
  • [24] Liver-Targeted Lentiviral Gene Therapy Achieves 100% of Normal Circulating FVIII Levels in Non-Human Primates
    Liu, Tongyao
    Cantore, Alessio
    Patarroyo-White, Sue
    Milani, Michela
    Biffi, Mauro
    Moffit, Jeff
    Drager, Douglas
    Naldini, Luigi
    Peters, Rob
    MOLECULAR THERAPY, 2019, 27 (04) : 307 - 307
  • [25] Development and application of a FIX-specific immunoassay to monitor hemophilia B gene therapy
    Weber, A.
    Engelmaier, A.
    Voelkel, D.
    Scheiflinger, F.
    Rottensteiner, H.
    JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2016, 14 : 50 - 50
  • [26] Combination Therapy to Enable AAV Re-Dosing and Mitigate Transgene Immunity in Liver-Targeted Gene Therapy
    Rana, Jyoti
    Kumar, Sandeep R. P.
    Munoz, Maite
    Yamada, Kentaro
    Bertollini, Thais
    Markusic, David M.
    Corti, Manuela
    Byrne, Barry J.
    Herzog, Roland W.
    Biswas, Moanaro
    BLOOD, 2022, 140
  • [27] RNA polymerase III-driven expression cassettes in human gene therapy
    Medina, MFC
    Joshi, S
    CURRENT OPINION IN MOLECULAR THERAPEUTICS, 1999, 1 (05) : 580 - 594
  • [28] Liver-targeted gene therapy by SV40-based vectors using the hydrodynamic injection method
    Arad, U
    Zeira, E
    Abd El-Latif, M
    Mukherjee, S
    Mitchell, L
    Pappo, O
    Galun, E
    Oppenheim, A
    HUMAN GENE THERAPY, 2005, 16 (03) : 361 - 371
  • [29] Liver-targeted gene therapy prevents chronic hypothermia and doubles survival in complex III deficient mice
    Banerjee, Rishi
    Purhonen, Janne
    Fellman, Vineta
    Kallijarvi, Jukka
    BIOCHIMICA ET BIOPHYSICA ACTA-BIOENERGETICS, 2024, 1865 : 99 - 99
  • [30] AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
    Crudele, Julie M.
    Finn, Jonathan D.
    Siner, Joshua I.
    Martin, Nicholas B.
    Niemeyer, Glenn P.
    Zhou, Shangzhen
    Mingozzi, Federico
    Lothrop, Clinton D., Jr.
    Arruda, Valder R.
    BLOOD, 2015, 125 (10) : 1553 - 1561
12345