STAAR, a phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Dose escalation phase results

被引：0

作者：

Hopkin, Robert J. ^{[1
]}

Ganesh, Jaya ^{[2
]}

Deegan, Patrick ^{[3
]}

Goker-Alpan, Ozlem ^{[4
]}

Bernat, John ^{[5
]}

Wilcox, William R. ^{[6
]}

Pahl, Madeleine V. ^{[7
]}

Whitley, Chester B. ^{[8
]}

Hughes, Derralynn ^{[9
]}

Nicholls, Kathleen ^{[10
]}

Cao, Liching ^{[11
]}

Chen, Michael ^{[11
]}

Shiue, Lisa H. ^{[11
]}

Bowden, Emma ^{[11
]}

Jaggumantri, Sravan ^{[11
]}

Passalacqua, Cristobal ^{[11
]}

Souberbielle, Bernard ^{[11
]}

Cockroft, Bettina M. ^{[11
]}

机构：

[1] Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA

[2] Icahn Sch Med Mt Sinai, New York, NY USA

[3] Addenbrookes Hosp, Cambridge, England

[4] Lysosomal & Rare Disorders Res & Treatment Ctr In, Fairfax, VA USA

[5] Univ Iowa, Iowa City, IA USA

[6] Emory Univ, Human Genet, Atlanta, GA USA

[7] Univ Calif Irvine, Orange, CA USA

[8] Univ Minnesota, Minneapolis, MN USA

[9] UCL, London, England

[10] Royal Melbourne Hosp, Parkville, Australia

[11] Sangamo Therapeut Inc, Brisbane, CA USA

来源：

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

关键词：

D O I：

10.1016/j.ymgme.2022.107152

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

169

引用

页码：61 / 62

页数：2

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