Safety and Increased Transduction Efficiency in the Adult Nonhuman Primate Central Nervous System with Intravenous Delivery of Two Novel Adeno-Associated Virus Capsids

被引:0
|
作者
Sah, Dinah [1 ]
Mazzarelli, Allyse [1 ]
Christensen, Emily [1 ]
Thompson, Jeff [1 ]
Lindgren, Holly [1 ]
Felix-Ortiz, Ada [1 ]
Aubin, Justin [1 ]
Scheel, Maria [1 ]
Pechan, Peter [1 ]
Horowitz, Eric [1 ]
Fine, Shamone [1 ]
Shu, Yanqun [1 ]
Zhou, Xiaochuan [1 ]
Gamba-Vitalo, Christina [1 ]
Carter, Todd [1 ]
Chen, Qingmin [1 ]
Carroll, Jenna [1 ]
Goulet, Martin [1 ]
Patzke, Holger [1 ]
机构
[1] Voyager Therapeut, Cambridge, MA USA
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
661
引用
收藏
页码:305 / 305
页数:1
相关论文
共 50 条
  • [31] An approach to identify novel Adeno-Associated Virus (AAV) capsids that provide enhanced retinal cell transduction following intravitreal injections
    Powers, John Michael
    Adachi, Kei
    Sivyer, Benjamin
    Taylor, William Rowland
    Pennesi, Mark E.
    Nakai, Hiroyuki
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2016, 57 (12)
  • [32] Biodistribution and safety profile of recombinant adeno-associated virus serotype 6 vectors following intravenous delivery
    Stone, Daniel
    Liu, Ying
    Li, Zong-Yi
    Strauss, Robert
    Finn, Eric E.
    Allen, James M.
    Chamberlain, Jeffrey S.
    Lieber, Andre
    JOURNAL OF VIROLOGY, 2008, 82 (15) : 7711 - 7715
  • [33] 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
  • [34] Understanding the Immune Response to Central Nervous System-Directed Adeno-Associated Virus Vectors
    Harkins, Ashley
    Tutto, Amanda
    Sylvia, Katelyn
    Blackwood, Meghan
    Arjomandnejad, Motahareh
    Kopec, Acadia
    Keeler, Allison
    MOLECULAR THERAPY, 2024, 32 (04) : 237 - 237
  • [35] Adeno-Associated Virus-Mediated Expression of Rituximab for the Treatment of Central Nervous System Lymphoma
    Werner, Marcela S.
    Cerqueira, Otto D.
    Abdullah, Lama
    Roamer, Jillian
    Greig, Jenny A.
    Wilson, James M.
    MOLECULAR THERAPY, 2024, 32 (04) : 827 - 827
  • [36] Characterization of the Immune Response to Adeno-Associated Virus Vectors in the Central Nervous System of an Ovine Model
    Yingling, Nathan K.
    Harkins, Ashley
    Benatti, Hector Ribeiro
    Blackwood, Meghan
    Arjomandnejad, Motahareh
    Taghian, Toloo
    Nath, Sarah
    Reil, Darcy
    Gray-Edwards, Heather L.
    Keeler-Klunk, Allison M.
    MOLECULAR THERAPY, 2024, 32 (04) : 834 - 835
  • [37] Pre-retinal delivery of recombinant adeno-associated virus vector significantly improves retinal transduction efficiency
    Zhang, Hanmeng
    Sajdak, Benjamin S.
    Merriman, Dana K.
    Carroll, Joseph
    Lipinski, Daniel M.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021, 22 : 96 - 106
  • [38] Safety Study of Adeno-Associated Virus Serotype 2-Mediated Human Acid Sphingomyelinase Expression in the Nonhuman Primate Brain
    Salegio, Ernesto A.
    Samaranch, Lluis
    Jenkins, Russell W.
    Clarke, Christopher J.
    Lamarre, Clementine
    Beyer, Janine
    Kells, Adrian P.
    Bringas, John
    San Sebastian, Waldy
    Richardson, R. Mark
    Rosenbluth, Kate H.
    Hannun, Yusuf A.
    Bankiewicz, Krystof S.
    Forsayeth, John
    HUMAN GENE THERAPY, 2012, 23 (08) : 891 - 902
  • [39] Assessment of transduction efficiency and tropism of different adeno-associated virus (AAV) serotypes based on route of delivery in rats
    Han, Ian C.
    Jiao, Chunhua
    Kaalberg, Emily E.
    Bhattarai, Sajag
    Burnight, Erin R.
    Tucker, Budd A.
    Mullins, Robert F.
    Stone, Edwin M.
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2018, 59 (09)
  • [40] Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice
    Hinderer, Christian
    Katz, Nathan
    Louboutin, Jean-Pierre
    Bell, Peter
    Yu, Hongwei
    Nayal, Mohamad
    Kozarsky, Karen
    O'Brien, W. Timothy
    Goode, Tamara
    Wilson, James M.
    HUMAN GENE THERAPY, 2016, 27 (11) : 906 - 915