First-In-Human Intrathecal Gene Transfer Study for Giant Axonal Neuropathy: Interim Analysis of Efficacy and Review of Safety

被引：0
|
作者：
Bharucha-Goebel, Diana [1 ]
Saade, Dimah [1 ]
Jain, Mina [2 ]
Waite, Melissa [2 ]
Norato, Gina [3 ]
Cheung, Ken [1 ]
Foley, Reghan [1 ]
Soldatos, Ariane [4 ]
Rybin, Denis [5 ]
Lehky, Tanya [6 ]
Hu, Ying [1 ]
Whitehead, Matthew [7 ]
Del Hoyo, Roberto Calcedo [8 ]
Jacobson, Steven [9 ]
Nath, Avindra [9 ]
Grieger, Joshua [10 ]
Samulski, Jude [11 ]
Gray, Steven [12 ]
Bonnemann, Carsten [1 ]
机构：
[1] NINDS, Neurogenet Branch, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[2] NINDS, Rehabil Dept, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[3] NINDS, Off Biostat, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[4] NINDS, Pediat Neurol, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[5] Pfizer Inc, Cambridge, MA USA
[6] NINDS, Neurophysiol, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[7] Childrens Natl Hlth Syst, Neuroradiol, Washington, DC USA
[8] Univ Penn, Philadelphia, PA 19104 USA
[9] NINDS, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA
[10] Asklepios Biopharmaceut Inc, Chapel Hill, NC USA
[11] Bamboo Therapeut, Chapel Hill, NC USA
[12] UT Southwestern, Pediat, Dallas, TX USA
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：
64
引用
收藏
页码：32 / 33
页数：2
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