First in-human intracisternal dosing of RGX-111 (adeno-associated virus 9/human α-L-iduronidase) for a 20-month-old child with mucopolysaccharidosis type I (MPS I): 1 year follow-up

被引：0
|
作者：
Wang, Raymond Y. [1 ]
Beydoun, Tammam [1 ]
Movsesyan, Nina [2 ]
Kan, Shih-Hsin [2 ]
Taylor, Mery [2 ]
Stockton, Winnie [2 ]
Nestrasil, Igor [3 ]
Pukenas, Bryan [4 ]
Falabella, Paulo [5 ]
Cho, Yoonjin [5 ]
Nevoret, Marie-Laure [5 ]
机构：
[1] CHOC Childrens Specialists, Orange, CA USA
[2] CHOC Childrens Hosp, Orange, CA USA
[3] Univ Minnesota, Minneapolis, MN USA
[4] Hosp Univ Penn, 3400 Spruce St, Philadelphia, PA 19104 USA
[5] REGENXBIO Inc, Rockville, MD USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2021年 / 132卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2020.12.270
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
264
引用
收藏
页码：S110 / S110
页数：1
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