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：

引用

页码：32 / 33

页数：2

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