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
收藏
页码：61 / 62
页数：2
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