Development of a Self-Complementary AAV.U7snRNA Vector for Efficient Dystrophin Exon 53 Skipping

被引：0
|
作者：
Qian, Randolph [1 ]
Foltz, Steven J. [1 ]
Jin, Lan [1 ]
Chen, Ting [1 ]
Qiao, Chunping [1 ]
Elliott, Kirk W. [1 ]
Hafiz, Ikram [1 ]
Yang, Jason [1 ]
Karumuthil-Melethil, Subha [1 ]
Bruder, Joseph T. [1 ]
Danos, Olivier F. [1 ]
Liu, Ye [1 ]
机构：
[1] REGENXBIO, Res Early Dev, Rockville, MD USA
来源：
MOLECULAR THERAPY | 2023年 / 31卷 / 04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：
1565
引用
收藏
页码：736 / 736
页数：1
相关论文
共 41 条
  • [21] Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
    Gushchina, Liubov, V
    Bradley, Adrienne J.
    Vetter, Tatyana A.
    Lay, Jacob W.
    Rohan, Natalie L.
    Frair, Emma C.
    Wein, Nicolas
    Flanigan, Kevin M.
    MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2023, 31
  • [22] Effect of combined treatment with soluble activin receptor IIB and AAV-U7-mediated dystrophin exon skipping on muscle function in mdx mice
    Hoogaars, W. M. H.
    Mouisel, E.
    Relizani, K.
    Pasternack, A.
    Hourde, C.
    Precigout, G.
    Ferry, A.
    Garcia, L.
    Kemaladewi, D. U.
    Aartsma-Rus, A.
    Ritvos, O.
    't Hoen, P. A. C.
    Amthor, H.
    NEUROMUSCULAR DISORDERS, 2011, 21 (9-10) : 703 - 703
  • [23] Characterization and Treatment of a Novel Mouse Model of Duchenne Muscular Dystrophy Using U7snRNA to Mediate Exon Skipping for the Second Calponin Homology Domain of Dystrophin
    Lesman, Daniel N.
    Li, Ding
    Rajakumar, Dhanarajan
    Al-Tabosh, Hayat
    Rafferty, Rachel
    Rodriguez, Yacidzohara
    Wein, Nicolas
    MOLECULAR THERAPY, 2021, 29 (04) : 214 - 214
  • [24] An exon-skipping strategy using AAV8-U7snRNA vectors for the treatment of Duchenne muscular dystrophy - results in GRMD dogs and clinical perspectives
    Le Guiner, Caroline
    Montus, Marie
    Servais, Laurent
    Garcia, Luis
    Fromes, Yves
    Hogrel, Jean-Yves
    Carlier, Pierre
    Cherel, Yan
    Moullier, Philippe
    Voit, Thomas
    HUMAN GENE THERAPY, 2012, 23 (10) : A25 - A25
  • [25] AAV9-U7snRNA Mediated Skipping of DMD Exon 2: Absence of Off-Target Splicing Effects as Demonstrated by RNA-Seq
    Flanigan, Kevin M.
    Wein, Nicolas
    Gushchina, Liubov
    Waldrop, Megan
    Weiss, Robert B.
    MOLECULAR THERAPY, 2019, 27 (04) : 377 - 377
  • [26] RNA-Seq shows an absence of off-target splicing effects in AAV9-U7snRNA mediated skipping of DMD exon 2
    Flanigan, K.
    Wein, N.
    Gushchina, L.
    Waldrop, M.
    Weiss, R.
    NEUROMUSCULAR DISORDERS, 2019, 29 : S89 - S89
  • [27] A single neonatal delivery of an exon 2 directed AAV9.U7snRNA vector results in long-term dystrophin expression that prevents pathologic features in the Dup2 mouse
    Wein, N.
    Simmons, T.
    Gumienny, F.
    Yurkoski, J.
    Huang, N.
    Muntoni, F.
    Flanigan, K.
    NEUROMUSCULAR DISORDERS, 2016, 26 : S126 - S127
  • [28] A Single Neonatal Delivery of an Exon 2 Directed AAV9.U7snRNA Vector Results in Long-Term Dystrophin Expression That Prevents Pathologic Features in the Dup2 Mouse
    Wein, Nicolas
    Simmons, Tabatha R.
    Gumienny, Felecia
    Yurkoski, Jacqueline
    Huang, Nianyuan
    Muntoni, Francesco
    Flanigan, Kevin M.
    MOLECULAR THERAPY, 2016, 24 : S247 - S248
  • [29] Dystrophin rescue in mdx mice after injection of AAV1 vectors harboring an engineered U7snRNA carrying antisense sequences against exon 23
    Goyenvalle, A
    Vulin, A
    Peccate, C
    Leturcq, F
    Kaplan, JC
    Danos, O
    Garcia, L
    NEUROMUSCULAR DISORDERS, 2004, 14 (8-9) : 610 - 611
  • [30] Absence of toxicity with intravenous dosing of the Exon 2-skipping AAV9.U7-ACCA vector in non-human primates
    Gushchina, L.
    Frair, E.
    Rohan, N.
    Bradley, A.
    Simmons, T.
    Chavan, H.
    Waldrop, M.
    Wein, N.
    Flanigan, K.
    NEUROMUSCULAR DISORDERS, 2020, 30 : S109 - S109
12345