A novel AAV-based miQURE gene therapy for SCA3

被引:0
|
作者
Toonen, L. J. A. [1 ]
Nobre, R. [2 ]
Haas, E. [3 ]
Huebener-Schmid, J. [3 ]
Martier, R. [1 ]
Valles, A. [1 ]
Higgens, J. J. [1 ]
van Deventer, S. [1 ]
Nguyen, H. P. [3 ]
Pereira de Almeida, L. [2 ]
Konstantinova, P. [1 ]
Evers, M. M. [1 ]
机构
[1] UniQure Biopharma BV, Amsterdam, Netherlands
[2] Univ Coimbra, Coimbra, Portugal
[3] Inst Med Genet & Appl Genom, Tubingen, Germany
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
P247
引用
收藏
页码:A91 / A92
页数:2
相关论文
共 50 条
  • [41] Development of a universal extraction protocol for multiple AAV-based gene therapy shedding assays
    Wang, Z.
    Philipp, I.
    Qiu, X.
    Weitz, S.
    Armand, T.
    Uzgiris, A.
    HUMAN GENE THERAPY, 2019, 30 (11) : A114 - A114
  • [42] AAV-based gene therapy approaches for genetic forms of tauopathies and related neurogenetic disorders
    Al Kabbani, Mohamed Aghyad
    Wunderlich, Gilbert
    Koeler, Christoph
    Zempel, Hans
    BIOCELL, 2022, 46 (04) : 847 - 853
  • [43] Preclinical Evaluation of an AAV-Based Gene Therapy Approach for the Treatment of Friedreich Ataxia Cardiomyopathy
    Martelli, Alain
    Assaf, Basel T.
    Gooch, Renea
    Ma, Tiffany
    Whiteley, Laurence O.
    Bulawa, Christine
    MOLECULAR THERAPY, 2019, 27 (04) : 51 - 51
  • [44] Cerebellomedullary Cistern Delivery for AAV-Based Gene Therapy: A Technical Note for Nonhuman Primates
    Samaranch, Lluis
    Bringas, John
    Pivirotto, Philip
    Sebastian, Waldy San
    Forsayeth, John
    Bankiewicz, Krystof
    HUMAN GENE THERAPY METHODS, 2016, 27 (01) : 13 - 16
  • [45] High Expression Bioengineered Factor VIII Enables AAV-Based Gene Therapy of Hemophilia A
    Spencer, H. Trent
    Brown, Harrison C.
    Shields, Jordan E.
    Wright, J. Fraser
    Doering, Christopher B.
    MOLECULAR THERAPY, 2013, 21 : S253 - S253
  • [46] Focused Update on AAV-Based Gene Therapy Clinical Trials for Inherited Retinal Degeneration
    Paula I. Fuller-Carter
    Hamed Basiri
    Alan R. Harvey
    Livia S. Carvalho
    BioDrugs, 2020, 34 : 763 - 781
  • [47] Suppression of Choroidal Neovascularization by AAV-Based Dual-Acting Antiangiogenic Gene Therapy
    Askou, Anne Louise
    Alsing, Sidsel
    Benckendorff, Josephine N. E.
    Holmgaard, Andreas
    Mikkelsen, Jacob Giehm
    Aagaard, Lars
    Bek, Toke
    Corydon, Thomas J.
    MOLECULAR THERAPY-NUCLEIC ACIDS, 2019, 16 : 38 - 50
  • [48] AAV-Based Gene Therapy in a Mouse Model of Smith Lemli Opitz Syndrome (SLOS)
    Pasta, Saloni Y.
    Ting, Flora
    Tabron, Dorothy
    Sun, Sean
    Kadakia, Saurin
    Shackleton, Cedric
    Watson, Gordon
    MOLECULAR THERAPY, 2015, 23 : S147 - S147
  • [49] AAV-based therapy for mitochondrial diseases and optic neuropathies
    Viscomi, Carlo
    Balmaceda, Valeria
    Corra, Samantha
    Cerutti, Raffaele
    BIOCHIMICA ET BIOPHYSICA ACTA-BIOENERGETICS, 2024, 1865 : 21 - 22
  • [50] AAV-based liver-targeted gene therapy for MNGIE: proposal for a clinical trial
    van den Ameele, J.
    Cutting, E.
    Marti, R.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A95 - A95