Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

被引:2
|
作者
McIntosh, Jenny H. [1 ]
Mohamed, Saira [1 ]
Ireland, Lydia [1 ]
Morton, Christopher L. [2 ]
Reiss, Ulrike M. [3 ]
Chowdary, Pratima [4 ,5 ]
Tuddenham, Edward G. D. [4 ,5 ]
Davidoff, Andrew M. [2 ]
Nathwani, Amit C. [1 ,6 ]
机构
[1] UCL, Inst Canc, London, England
[2] St Jude Childrens Res Hosp, Dept Surg, 332 N Lauderdale St, Memphis, TN 38105 USA
[3] St Jude Childrens Res Hosp, Dept Hematol, 332 N Lauderdale St, Memphis, TN 38105 USA
[4] UCL, London, England
[5] Royal Free Hosp, Katharine Dormandy Haemophilia & Thrombosis Ctr, London, England
[6] Royal Free London NHS Fdn Trust, London, England
关键词
D O I
10.1182/blood-2023-188383
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页数:4
相关论文
共 50 条
  • [31] Improving brain delivery of adeno-associated viral gene therapy vectors for the treatment of MPS IIIC
    O'Leary, Claire
    Forte, Gabriella
    Mitchell, Nadia
    Youshani, Amir Saam
    Wellby, Martin
    Russell, Katharina
    Palmer, David
    Henckaerts, Els
    Asl, Ian Kamaly
    Bigger, Brian
    MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S111 - S111
  • [32] Synthetic Biology: Emerging Concepts to Design and Advance Adeno-Associated Viral Vectors for Gene Therapy
    Wagner, Hanna J.
    Weber, Wilfried
    Fussenegger, Martin
    ADVANCED SCIENCE, 2021, 8 (09)
  • [33] Autophagy Determines Efficiency of Liver-Directed Gene Therapy With Adeno-Associated Viral Vectors
    Hoesel, Marianna
    Huber, Anke
    Bohlen, Susanne
    Lucifora, Julie
    Ronzitti, Giuseppe
    Puzzo, Francesco
    Boisgerault, Florence
    Hacker, Ulrich T.
    Kwanten, Wilhelmus J.
    Kloeting, Nora
    Blueher, Matthias
    Gluschko, Alexander
    Schramm, Michael
    Utermoehlen, Olaf
    Bloch, Wilhelm
    Mingozzi, Federico
    Krut, Oleg
    Buening, Hildegard
    HEPATOLOGY, 2017, 66 (01) : 252 - 265
  • [34] Retinal Gene Therapy Using Adeno-Associated Viral Vectors: Multiple Applications for a Small Virus
    Hauswirth, William W.
    HUMAN GENE THERAPY, 2014, 25 (08) : 671 - 678
  • [35] Construction of MDR1 adeno-associated virus vectors for gene therapy
    Baudard, M
    ABC TRANSPORTERS: BIOCHEMICAL, CELLULAR, AND MOLECULAR ASPECTS, 1998, 292 : 538 - 545
  • [36] Adeno-associated viral vector integration: implications for long-term efficacy and safety
    Batty, Paul
    Lillicrap, David
    JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2024, 22 (11) : 2945 - 2960
  • [37] Bat adeno-associated viruses as gene therapy vectors with the potential to evade human neutralizing antibodies
    Ya Li
    Jialu Li
    Yunbo Liu
    Zhengli Shi
    Haizhou Liu
    Yuquan Wei
    Lin Yang
    Gene Therapy, 2019, 26 : 264 - 276
  • [38] Bat adeno-associated viruses as gene therapy vectors with the potential to evade human neutralizing antibodies
    Li, Ya
    Li, Jialu
    Liu, Yunbo
    Shi, Zhengli
    Liu, Haizhou
    Wei, Yuquan
    Yang, Lin
    GENE THERAPY, 2019, 26 (06) : 264 - 276
  • [39] Bat Adeno-Associated Viruses as Gene Therapy Vectors with the Potential to Evade Human Neutralizing Antibodies
    Li, Ya
    Liu, Yunbo
    Shi, Zhengli
    Liu, Haizhou
    Wei, Yuquan
    Yang, Lin
    MOLECULAR THERAPY, 2019, 27 (04) : 341 - 341
  • [40] In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model
    Mattar, Citra N. Z.
    Gil-Farina, Irene
    Rosales, Cecilia
    Johana, Nuryanti
    Tan, Yvonne Yi Wan
    McIntosh, Jenny
    Kaeppel, Christine
    Waddington, Simon N.
    Biswas, Arijit
    Choolani, Mahesh
    Schmidt, Manfred
    Nathwani, Amit C.
    Chan, Jerry K. Y.
    MOLECULAR THERAPY, 2017, 25 (08) : 1843 - 1853