Development of an AAV9-mediated gene therapy for hereditary spastic paraplegia 47

被引：0
|
作者：
Scarrott, J. M. [1 ]
Coldicott, I. [1 ]
Davies, A. [2 ]
Hirst, J. [2 ]
Karyka, E. [1 ]
Bauer, C. [1 ]
Azzouz, M. [1 ]
机构：
[1] Univ Sheffield, Sheffield Inst Translat Neurosci, Sheffield, S Yorkshire, England
[2] Univ Cambridge, Cambridge Inst Med Res, Cambridge, England
来源：
HUMAN GENE THERAPY | 2019年 / 30卷 / 08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：
P03
引用
收藏
页码：A18 / A18
页数：1
相关论文
共 50 条
  • [31] Hereditary spastic paraplegia caused by a mutation in the VCP gene
    de Bot, Susanne T.
    Schelhaas, Helenius J.
    Kamsteeg, Erik-Jan
    van de Warrenburg, Bart P. C.
    [J]. BRAIN, 2012, 135
  • [32] Viral vector-mediated gene therapy for hereditary spastic paraplegia n.15 (HSP15)
    Pulix, M.
    Whiteley, L.
    Azzouz, M.
    [J]. HUMAN GENE THERAPY, 2019, 30 (08) : A27 - A28
  • [33] Gene Therapy Approach for Hereditary Spastic Paraplegia Type 52 (SPG52)
    Rodriguez-Estevez, Laura
    Almolda, Beatriz
    Fernandez-Carasa, Irene
    Edo, Angel
    Sanchez, Angela
    Brao, Alejandro
    Bosch, Assumpcio
    Consiglio, Antonella
    Chillon, Miguel
    [J]. MOLECULAR THERAPY, 2023, 31 (04) : 419 - 419
  • [34] Mutation in the AP4B1 gene cause hereditary spastic paraplegia type 47 (SPG47)
    Peter Bauer
    Esther Leshinsky-Silver
    Lubov Blumkin
    Nina Schlipf
    Christopher Schröder
    Julia Schicks
    Dorit Lev
    Olaf Riess
    Tally Lerman-Sagie
    Ludger Schöls
    [J]. neurogenetics, 2012, 13 : 73 - 76
  • [35] Mutation in the AP4B1 gene cause hereditary spastic paraplegia type 47 (SPG47)
    Bauer, Peter
    Leshinsky-Silver, Esther
    Blumkin, Lubov
    Schlipf, Nina
    Schroeder, Christopher
    Schicks, Julia
    Lev, Dorit
    Riess, Olaf
    Lerman-Sagie, Tally
    Schoels, Ludger
    [J]. NEUROGENETICS, 2012, 13 (01) : 73 - 76
  • [36] Prevention of cardiac function in mdx mice after AAV9-mediated microdystrophin gene transfer
    Schinkel, S.
    Bauer, R.
    Rutschow, D.
    Stucka, R.
    Lochmueller, H.
    Kleinschmidt, J.
    Katus, H.
    Mueller, O.
    [J]. HUMAN GENE THERAPY, 2010, 21 (10) : 1377 - 1377
  • [37] AAV9-mediated gene transfer of desmin ameliorates cardiomyopathy in desmin-deficient mice
    Heckmann, M. B.
    Bauer, R.
    Jungmann, A.
    Winter, L.
    Rapti, K.
    Strucksberg, K-H
    Clemen, C. S.
    Li, Z.
    Schroeder, R.
    Katus, H. A.
    Mueller, O. J.
    [J]. GENE THERAPY, 2016, 23 (8-9) : 673 - 679
  • [38] AAV9-mediated gene transfer of desmin ameliorates cardiomyopathy in desmin-deficient mice
    M B Heckmann
    R Bauer
    A Jungmann
    L Winter
    K Rapti
    K-H Strucksberg
    C S Clemen
    Z Li
    R Schröder
    H A Katus
    O J Müller
    [J]. Gene Therapy, 2016, 23 : 673 - 679
  • [39] AAV9-Mediated Gene Therapy for NGLY1 Deficiency and Assessment of GNA Biomarker Changes in a Rat Disease Model
    Mueller, William F.
    Zhu, Lei
    Dwight, Selina S.
    Beahm, Brendan
    Lee, Kevin J.
    Wilsey, Matt
    Mak, Justin
    Pollard, Laura
    Wood, Tim
    Cowan, Tina
    Crawford, Brett
    Wechsler, Thomas
    [J]. MOLECULAR THERAPY, 2020, 28 (04) : 124 - 125
  • [40] Functional analysis of the hereditary spastic paraplegia gene Atlastin in flies
    Martinuzzi, Andrea
    Orso, Genny
    Tosetto, Jessica
    Stefani, Stefania
    Daga, Andrea
    [J]. ANNALS OF NEUROLOGY, 2006, 60 : S54 - S54
12345