A Human-Ready Regulated AAV9/miniMECP2-miRARE Gene Therapy (TSHA-102) Improves Survival, Weight, and Behavior after Intracerebroventricular (ICV) Dosing in the Neonatal Knockout Rett (RTT) Mouse Model

被引:0
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作者
Okposo, Kome [1 ]
Haque, Emdadul [1 ]
Sadhu, Chanchal [2 ]
Prasad, Suyash [2 ]
Newman, Mary [2 ]
Lawlor, Michael W. [3 ]
Gray, Steven J. [4 ]
Sinnett, Sarah E. [4 ]
机构
[1] Taysha Gene Therapies, Dept Res & Dev, Dallas, TX USA
[2] Taysha Gene Therapies, Dept Res & Dev, Dallas, TX USA
[3] Diverge Translat Sci Lab, Milwaukee, WI USA
[4] Univ Texas SouthWestern Med Ctr, Dept Pediat, Dallas, TX USA
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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
1365
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页码:644 / 644
页数:1
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