High level expression of human iduronidase throughout the brain in a murine model of mucopolysaccharidosis type I (MPS I) after non-invasive AAV-mediated gene delivery to the CNS

被引：0
|
作者：
Kozarsky, Karen [1 ]
Nan, Zhenhong [2 ]
Buckvold, Megan [2 ]
Karlen, Andrea [2 ]
Kitto, Kelley [2 ]
Podetz-Pedersen, Kelly M. [2 ]
Fairbanks, Carolyn [2 ]
Hanson, Leah R. [3 ]
Orchard, Paul [2 ]
Tolar, Jakub [2 ]
Frey, William H., II [3 ]
Low, Walter C. [2 ]
McIvor, R. Scott [2 ]
Belur, Lalitha [2 ]
机构：
[1] ReGenX Biosci, Washington, DC 20006 USA
[2] Univ Minnesota, Minneapolis, MN 55455 USA
[3] Reg Hosp, St Paul, MN 55101 USA
来源：
HUMAN GENE THERAPY | 2014年 / 25卷 / 11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：
OR052
引用
收藏
页码：A42 / A42
页数：1
相关论文
共 23 条
  • [1] High Level Expression of Human Iduronidase Throughout the Brain in a Murine Model of Mucopolysaccharidosis Type I After Non-Invasive AAV-Mediated Gene Delivery to the CNS
    Belur, Lalitha
    Nan, Zhenhong
    Buckvold, Megan
    Karlen, Andrea
    Kitto, Kelley
    Podetz-Pedersen, Kelly M.
    Fairbanks, Carolyn
    Hanson, Leah
    Tolar, Jakub
    Kozarsky, Karen
    Frey, William H., II
    Low, Walter C.
    McIvor, R. Scott
    MOLECULAR THERAPY, 2014, 22 : S236 - S236
  • [2] Intranasal Gene Delivery of AAV Iduronidase: An Effective and Non-Invasive Approach for Treatmentof CNS Disease in a Murine Model of MPS Type I
    Belur, Lalitha
    Buckvold, Megan
    Podetz-Pedersen, Kelly M.
    Riedl, Maureen
    Vulchanova, Lucy
    Hanson, Leah
    Fairbanks, Carolyn
    Kozarsky, Karen
    Frey, William H., II
    Low, Walter C.
    McIvor, R. Scott
    MOLECULAR THERAPY, 2016, 24 : S12 - S12
  • [3] Therapeutic Effectiveness of AAV-Mediated Iduronidase Delivery to the CNS Following Intravenous Administration in a Murine Model of Mucopolysaccharidosis Type I
    Belur, Lalitha
    Thuy An Tran
    Pedersen, Kelly M.
    Riedl, Maureen
    Vulchanova, Lucy
    Kozarsky, Karen
    Low, Walter C.
    McIvor, R. Scott
    MOLECULAR THERAPY, 2016, 24 : S148 - S148
  • [4] AAV Vector-Mediated Iduronidase Gene Delivery in a Murine Model of Mucopolysaccharidosis Type I: Comparing Different Routes of Delivery to the CNS
    Belur, Lalitha
    Nan, Zhenhong
    Podetz-Pedersen, Kelly M.
    Fairbanks, Carolyn A.
    Hanson, Leah R.
    Frey, William H.
    Kozarsky, Karen
    Low, Walter C.
    McIvor, R. Scott
    MOLECULAR THERAPY, 2013, 21 : S140 - S140
  • [5] Intranasal gene delivery of AAV9 iduronidase: A non-invasive and effective gene therapy approach for prevention of neurologic disease in a murine model of mucopolysaccharidosis type I
    Belur, Lalitha
    Buckvold, Megan
    Podetz-Pedersen, Kelly M.
    Riedl, Maureen
    Vulchanova, Lucy
    Hanson, Leah R.
    Fairbanks, Carolyn
    Kozarsky, Karen
    Freyll, William H.
    Low, Walter C.
    McIvor, R. Scott
    MOLECULAR GENETICS AND METABOLISM, 2016, 117 (02) : S26 - S26
  • [6] AAV-Mediated Phenotypic Correction of the CNS Manifestations of a Mouse Model of Mucopolysaccharidosis Type I
    Van de Graaf, Benjamin G.
    Wang, Marlene
    Kwaa, Abena
    Hackett, Neil R.
    Crystal, Ronald G.
    Sondhi, Dolan
    MOLECULAR THERAPY, 2014, 22 : S146 - S147
  • [7] Direct Administration of an AAV Vector Encoding Human α-L-Iduronidase to the CNS in a Murine Model of Mucopolysaccharidosis Type I
    Wolf, Daniel A.
    Nan, Zhenhong
    Swanson, Debra L.
    Koniar, Brenda
    Belur, Lalitha R.
    Whitley, Chester B.
    Low, Walter C.
    McIvor, R. Scott
    MOLECULAR THERAPY, 2009, 17 : S342 - S342
  • [8] Non-Invasive Intranasal Administration of AAV9-Iduronidase Prevents Emergence of Neurologic Disease and Neurocognitive Dysfunction in a Murine Model of Mucopolysaccharidosis Type I
    Belur, Lalitha
    Buckvold, Megan
    Podetz-Pedersen, Kelly
    Riedl, Maureen
    Vulchanova, Lucy
    Hanson, Leah R.
    Kozarsky, Karen
    Frey, William H.
    Low, Walter C.
    Fairbanks, Carolyn
    McIvor, R. Scott
    MOLECULAR THERAPY, 2015, 23 : S281 - S281
  • [9] Intrathecal AAV9-Mediated Gene Delivery Corrects Lysosomal Storage Throughout the CNS in a Large Animal Model of Mucopolysaccharidosis Type I
    Hinderer, Christian
    Wang, Thomas
    Gurda, Brittney
    Bell, Peter
    Sikora, Tracey
    Bagel, Jessica
    Haskins, Mark
    Wilson, James M.
    MOLECULAR THERAPY, 2014, 22 : S233 - S234
  • [10] Gene delivery and biodistribution following intravenous administration of AAV9/rh10 iduronidase in a murine model of MPS type I
    Belur, Lalitha
    Thuy An Tran
    Podetz-Pedersen, Kelly M.
    Riedl, Maureen
    Vulchanova, Lucy
    Kozarsky, Karen
    Low, Walter C.
    McIvor, R. Scott
    MOLECULAR GENETICS AND METABOLISM, 2016, 117 (02) : S26 - S26
123