BB-301: A Single "Silence and Replace" AAV-Based Vector for the Treatment of Oculopharyngeal Muscular Dystrophy (OPMD)

被引：0
|
作者：
Strings-Ufombah, V. [1 ]
Malerba, A. [2 ]
Harbaran, S. [1 ]
Roth, F. [3 ]
Kao, S-C [1 ]
Cappellari, O. [2 ]
Nguyen, N. [2 ]
Takahashi, K. [1 ]
Kloth, C. [1 ]
Roelvink, P. [1 ]
Dickson, G. [2 ]
Trollet, C. [3 ]
Suhy, D. [1 ]
机构：
[1] Benitec Biopharma, Hayward, CA USA
[2] Royal Holloway Univ London, Sch Biol Sci, Egham, Surrey, England
[3] UPMC Paris, Sorbonne Univ, Inst Myol, Paris, France
来源：
MOLECULAR THERAPY | 2018年 / 26卷 / 05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：
354
引用
收藏
页码：165 / 165
页数：1
相关论文
共 9 条
  • [1] BB-301: a single "silence and replace" AAV-based vector for the treatment of oculopharyngeal muscular dystrophy (OPMD)
    Srtrings-ufombah, V.
    Malerba, A.
    Harbaran, S.
    Roth, F.
    Cappellari, O.
    Nguyen, N.
    Kao, S. C.
    Takahashi, K.
    Kloth, C.
    Poelvink, P.
    Dickson, G.
    Suhy, D.
    Trollet, C.
    HUMAN GENE THERAPY, 2018, 29 (12) : A142 - A143
  • [2] BB-301: a single "silence and replace" AAV-based vector for the treatment of oculopharyngeal muscular dystrophy
    Strings-Ufombah, V.
    Malerba, A.
    Harbaran, S.
    Roth, F.
    Cappellari, O.
    Nguyen, N.
    Kao, S.
    Takahashi, K.
    Kloth, C.
    Roelvink, P.
    Dickson, G.
    Trollet, C.
    Suhy, D.
    NEUROMUSCULAR DISORDERS, 2018, 28 : S140 - S140
  • [3] BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy
    Strings-Ufombah, Vanessa
    Malerba, Alberto
    Kao, Shih-Chu
    Harbaran, Sonal
    Roth, Fanny
    Cappellari, Ornella
    Lu-Nguyen, Ngoc
    Takahashi, Keiko
    Mukadam, Sophie
    Kilfoil, Georgina
    Kloth, Claudia
    Roelvink, Petrus
    Dickson, George
    Trollet, Capucine
    Suhy, David
    MOLECULAR THERAPY-NUCLEIC ACIDS, 2021, 24 : 67 - 78
  • [4] "Silence and Replace" : Development of a single AAV vector system for the treatment of oculopharyngeal muscular dystrophy (OPMD)
    Strings, V.
    Malerba, A.
    Harbaran, S.
    Cappellari, O.
    Roth, F.
    Chaytow, H.
    Cordova, G.
    Trollet, C.
    Dickson, G.
    Roelvink, P.
    Suhy, D.
    HUMAN GENE THERAPY, 2017, 28 (12) : A8 - A8
  • [5] AXO-AAV-OPMD: Proof-Of-Concept for a Single-Vector Silence and Replace Gene Therapy for the Treatment of Oculopharyngeal Muscular Dystrophy
    Strings-Ufombah, Vanessa
    Corcoran, Gavin
    Korner, Paul
    Wright, Fraser
    Suhy, David
    MOLECULAR THERAPY, 2019, 27 (04) : 376 - 376
  • [6] TOWARDS DEVELOPMENT OF A 'SILENCE AND REPLACE' BASED APPROACH FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCULAR DYSTROPHY
    Suhy, David
    Malerba, Alberto
    Klein, Pierre
    Jarmin, Susan
    Bachtarzi, Houria
    Ferry, Arnaud
    Butler-Browne, Gillian
    Strings-Ufombah, Vanessa
    Graham, Michael
    Trollet, Capucine
    Dickson, George
    JOURNAL OF GENE MEDICINE, 2018, 20 (01):
  • [7] Intranuclear inclusion body (INI) pathology and muscle atrophy in oculopharyngeal muscular dystrophy can be reversed by "silence and replace'' AAV-mediated gene therapy
    Malerba, A.
    Klein, P.
    Lu-Nguyen, N.
    Cappellari, O.
    Strings-Ufombah, V.
    Harbaran, S.
    Roelvink, P.
    Suhy, D.
    Trollet, C.
    Dickson, G.
    HUMAN GENE THERAPY, 2018, 29 (12) : A137 - A137
  • [8] PPMO pre-treatment is beneficial for AAV-based gene therapy in Duchenne muscular dystrophy
    Forand, A.
    Muchir, A.
    Mougenot, N.
    Sevoz-Couche, C.
    Peccate, C.
    Lemaitre, M.
    Wood, M.
    Lorain, S.
    Pietri-Rouxel, F.
    NEUROMUSCULAR DISORDERS, 2019, 29 : S164 - S165
  • [9] Novel Single AAV Vector Treatment for Congenital Muscular Dystrophy Type 1A (MDC1A) Using CRISPR-GNDM® Technology
    Qin, Yuanbo
    Akbulut, Talha
    Mandraju, Rajakumar
    Connolly, Keith
    Pranami, Payal
    Foster, Claudia
    Chezhiyan, Vandhana
    Shottek, Alison
    Yamagata, Tetsuya
    MOLECULAR THERAPY, 2022, 30 (04) : 16 - 16
1