AAV.U7.ex44 mediates efficient exon skipping, protein restoration & phenotype rescue - pre-clinical intramuscular and dose escalation study for a mutational hotspot of the Duchenne muscular dystrophy (DMD)

被引：0
|
作者：
Rajakumar, D. [1 ]
Almeida, C. [1 ]
Atre, C. [1 ]
Lesman, D. [1 ]
Li, D. [1 ]
Rodriguez, Y. [1 ]
Young, C. [2 ]
Spencer, M. [2 ]
Flanigan, K. [1 ]
Wein, N. [1 ]
机构：
[1] Nationwide Childrens Hosp, Columbus, OH USA
[2] Univ Calif Los Angeles, Los Angeles, CA USA
来源：
NEUROMUSCULAR DISORDERS | 2024年 / 43卷
关键词：
D O I：
暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
701P
引用
收藏
页数：1
相关论文
共 5 条
  • [1] AAV.U7.ex44 Mediates Efficient Exon Skipping, Protein Restoration & Phenotype Rescue -Pre-Clinical Intramuscular & Dose Escalation Study for a Mutational Hotspot of the Duchenne Muscular Dystrophy (DMD)
    Rajakumar, Dhanarajan
    Atre, Chaitrali
    Li, Ding
    Mahadevan, Archana
    Young, Courtney
    Spencer, Melissa J.
    Vetter, Tatyana
    Flanigan, Kevin M.
    Wein, Nicolas
    MOLECULAR THERAPY, 2022, 30 (04) : 248 - 248
  • [2] AAV.U7 Induced Exon Skipping for a Mutational Hotspot (∼6%) of the DMD Gene Results in Efficient Exon Skipping, Protein Restoration and Force Improvement
    Rajakumar, Dhanarajan
    Lesman, Daniel
    Li, Ding
    Gaffney, Caleb
    Rafferty, Rachel
    Huang, Nianyuan
    Rodriguez, Yacidzohara
    Young, Courtney
    Spencer, Melissa
    Flanigan, Kevin
    Wein, Nicolas
    MOLECULAR THERAPY, 2020, 28 (04) : 463 - 463
  • [3] In Vivo Delivery of AAV.U7 Induce Efficient Exon Skipping for a Mutational Hotspot of the DMD Gene Results in Protein Restoration & Force Improvement in Skeletal Muscles, Heart & Diaphragm
    Rajakumar, Dhanarajan
    Li, Ding
    Lesman, Daniel
    Gaffney, Caleb
    Rodriguez, Yacidzohara
    Young, Courtney
    Spencer, Melissa J.
    Flanigan, Kevin M.
    Wein, Nicolas
    MOLECULAR THERAPY, 2021, 29 (04) : 126 - 126
  • [4] Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
    Simmons, Tabatha R.
    Vetter, Tatyana A.
    Huang, Nianyuan
    Vulin-Chaffiol, Adeline
    Wein, Nicolas
    Flanigan, Kevin M.
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021, 21 : 325 - 340
  • [5] AAV.U7 technology for two mutational hotspots of the DMD gene (∼6%) results in efficient exon skipping, protein restoration and force improvement
    Wein, N.
    Simmons, T.
    Rajakumar, D.
    Lesman, D.
    Li, D.
    Gaffney, C.
    Rafferty, R.
    Huang, N.
    Rodriguez, Y.
    Young, C.
    Spencer, M.
    Flanigan, K.
    NEUROMUSCULAR DISORDERS, 2020, 30 : S107 - S107
1