Phase 1/2 trial of AXO-AAV-GM1 (AAV9-GLB1) gene therapy for infantile- and juvenile-onset GM1 gangliosidosis

被引：0

作者：

Tifft, Cynthia ^{[1
]}

D'Souza, Precilla ^{[1
]}

Johnston, Jean M. ^{[1
]}

Acosta, Maria T. ^{[1
]}

Nicoli, Elena-Raluca ^{[1
]}

Rothermel, Caroline ^{[1
]}

Thurm, Audrey ^{[2
]}

Karunakara, Ajith ^{[3
]}

Thorp, Benjamin ^{[3
]}

Ross, Peter ^{[3
]}

Jameson, John ^{[3
]}

Sheehan, Michael ^{[3
]}

Vaughn, Toby ^{[3
]}

Valencia, Donna ^{[3
]}

De Boever, Erika ^{[3
]}

Corcoran, Gavin ^{[3
]}

机构：

[1] Nat Human Genome Res Inst, Bethesda, MD USA

[2] Nat Inst Mental Hlth, Bethesda, MD USA

[3] Sio Gene Therapies Inc, New York, NY USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2022年 / 135卷 / 02期

关键词：

D O I：

10.1016/j.ymgme.2021.11.317

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

302

引用

页码：S119 / S119

页数：1

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