Sustained improvement of clinical CNS and somatic features of Gaucher disease type 3 after haematopoietic stem cell (HSC) gene therapy: A first-in-world report

被引：1

作者：

Donald, Aimee ^{[1
]}

Potter, Jane ^{[2
]}

Church, Heather J. ^{[3
]}

Amirloo, Bahareh ^{[4
]}

Cooper, James ^{[5
]}

Egerton, Christine ^{[5
]}

Watts, Kelly E. ^{[6
]}

Hutton, Rebekah A. ^{[7
]}

Rust, Stewart ^{[8
]}

Bromley, Rebecca ^{[9
]}

Stivaros, Stavros M. ^{[10
]}

Adera, Mathews ^{[11
]}

Ridha, Essra ^{[11
]}

Veselinovic, Milena ^{[12
]}

Jones, Simon A. ^{[13
]}

Wynn, Robert ^{[14
]}

Broomfield, Alexander ^{[15
]}

机构：

[1] Univ Manchester, Fac Biol Med & Hlth, Manchester, England

[2] Royal Manchester Childrens Hosp, Manchester, England

[3] Manchester Univ NHS Fdn Trust, St Marys Hosp, Manchester Ctr Genom Med, Willink Biochem Genet Unit, Manchester, England

[4] Manchester Univ NHS Fdn Trust, Pharm, Manchester, England

[5] Manchester Fdn Trust, Willink Biochem Ctr, St Marys Hosp, Manchester, England

[6] Manchester Fdn Trust, R&I, Manchester, England

[7] St Marys Hosp, Willink Unit Metabol, Manchester, England

[8] Royal Manchester Childrens Hosp, Psychol, Manchester, England

[9] Univ Manchester, Manchester, England

[10] Royal Manchester Childrens Hosp, Radiol, Manchester, England

[11] Avrobio, Cambridge, MA USA

[12] AVROBIO, Clin Dev, Cambridge, MA USA

[13] St Marys Hosp, Manchester Ctr Genom Med, Manchester, England

[14] Royal Manchester Childrens Hosp, Stem Cell Transplant, Manchester, England

[15] Royal Manchester Childrens Hosp, Biochem Genet, Manchester, England

来源：

MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2022.107083

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

100

引用

页码：36 / 37

页数：2