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

页码：68 / 69

页数：2

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[1] Clinical outcomes and sustained biochemical engraftment following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA
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[2] Biochemical Engraftment and Clinical Outcomes Following Ex-Vivo Autologous Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA
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Buckland, Karen
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Rust, Stewart
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[3] BIOCHEMICAL ENGRAFTMENT AND CLINICAL OUTCOMES OF EX-VIVO AUTOLOGOUS STEM CELL GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS TYPE IIIA
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[5] Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial
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[6] Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress-NCT04201405
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