Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System

被引：5
|
作者：
McElroy, Abigail [1 ,2 ]
Sena-Esteves, Miguel [1 ,2 ,3 ]
Arjomandnejad, Motahareh [1 ,2 ]
Keeler, Allison M. M. [1 ,2 ,4 ]
Gray-Edwards, Heather L. L. [1 ,2 ,5 ,6 ,7 ]
机构：
[1] UMass Chan Med Sch, Horae Gene Therapy Ctr, Worcester, MA USA
[2] UMass Chan Med Sch, Li Weibo Inst Rare Dis Res, Worcester, MA USA
[3] UMass Chan Med Sch, Dept Neurol, Worcester, MA USA
[4] UMass Chan Med Sch, Dept Pediat, Worcester, MA USA
[5] UMass Chan Med Sch, Dept Radiol, Worcester, MA USA
[6] UMass Chan Med Sch, Horae Gene Therapy Ctr, 368 Plantat St, Worcester, MA 01605 USA
[7] UMass Chan Med Sch, Li Weibo Inst Rare Dis Res, 368 Plantat St, Worcester, MA 01605 USA
来源：
HUMAN GENE THERAPY | 2022年 / 33卷 / 17-18期
关键词：
central nervous system; gene therapy; AAV; NEUTRALIZING ANTIBODIES; TRANSGENE EXPRESSION; SKELETAL-MUSCLE; ADULT MICE; BRAIN; AAV; VECTORS; DELIVERY; TURNOVER; ROUTES;
D O I：
10.1089/hum.2022.170
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：
[No abstract available]
引用
收藏
页码：889 / 892
页数：4
相关论文
共 50 条
  • [1] Adeno-Associated Virus Vector for Central Nervous System Gene Therapy
    Zhu, Danqing
    Schieferecke, Adam J.
    Lopez, Paola A.
    Schaffer, David, V
    TRENDS IN MOLECULAR MEDICINE, 2021, 27 (06) : 524 - 537
  • [2] Gene transfer by adeno-associated virus vectors into the central nervous system
    Xiao, X
    Li, J
    McCown, TJ
    Samulski, RJ
    EXPERIMENTAL NEUROLOGY, 1997, 144 (01) : 113 - 124
  • [3] Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia
    May, Francis J.
    Head, PamelaSara E.
    Venturoni, Leah E.
    Chandler, Randy J.
    Venditti, Charles P.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021, 21 : 765 - 776
  • [4] Systemic gene delivery to the central nervous system using Adeno-associated virus
    Bourdenx, Mathieu
    Dutheil, Nathalie
    Bezard, Erwan
    Dehay, Benjamin
    FRONTIERS IN MOLECULAR NEUROSCIENCE, 2014, 7
  • [5] Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors
    Macdonald, Josephine
    Marx, Jennifer
    Buning, Hildegard
    HUMAN GENE THERAPY, 2021, 32 (19-20) : 1096 - 1119
  • [6] Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects
    Slyk, Zaneta
    Stachowiak, Natalia
    Malecki, Maciej
    BIOMEDICINES, 2024, 12 (07)
  • [7] Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders
    Daci, Rrita
    Flotte, Terence R.
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2024, 25 (02)
  • [8] My Pathway to Adeno-Associated Virus and Adeno-Associated Virus Gene Therapy: A Personal Perspective
    Carter, Barrie J.
    HUMAN GENE THERAPY, 2020, 31 (9-10) : 494 - 498
  • [9] Adeno-associated virus and lentivirus vectors: a refined toolkit for the central nervous system
    Blessing, Daniel
    Deglon, Nicole
    CURRENT OPINION IN VIROLOGY, 2016, 21 : 61 - 66
  • [10] Understanding the immune response to adeno-associated virus vectors in the central nervous system
    Harkins, A. L.
    Tutto, A.
    Sylvia, K.
    Arjomandnejad, M.
    Kopec, A. L.
    Keeler, A. M.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A14 - A15
12345