Sustained biochemical engraftment and early clinical outcomes following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA

被引：2
|
作者：
Jones, Simon [1 ]
Kinsella, Jane [2 ]
Holley, Rebecca [3 ]
Potter, Jane [2 ]
Booth, Claire [4 ]
Buckland, Karen [4 ]
Rust, Stewart [5 ]
Church, Heather J. [6 ]
Brammeier, Kathryn L. [7 ]
Lee, Helena [8 ]
Ford, Laura [8 ]
Ellison, Stuart M. [9 ]
Bigger, Brian [3 ]
Wynn, Robert [10 ]
机构：
[1] St Marys Hosp, Manchester, Lancs, England
[2] Royal Manchester Childrens Hosp, Manchester, Lancs, England
[3] Univ Manchester, Manchester, Lancs, England
[4] UCL, Inst Child Hlth, London, England
[5] Royal Manchester Childrens Hosp, Paediat Neuropsychol, Manchester, Lancs, England
[6] Manchester Univ NHS Fdn Trust, St Marys Hosp, Willink Biochem Genet Unit, Manchester Ctr Genom Med, Manchester, Lancs, England
[7] Manchester Univ NHS Fdn Trust, Manchester Ctr Genom Med, Manchester, Lancs, England
[8] St Marys Hosp, Manchester Ctr Genom Med, Manchester, Lancs, England
[9] Univ Manchester, Stemcell & Neurotherapies Grp, Manchester, Lancs, England
[10] Royal Manchester Childrens Hosp, Bone Marrow Transplantat Unit, Manchester, Lancs, England
来源：
MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2022.107171
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
188
引用
收藏
页码：68 / 69
页数：2
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