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

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

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Bibliographic Details
Published in:Molecular genetics and metabolism Vol. 135; no. 2; p. S119
Main Authors: Tifft, Cynthia, D’Souza, Precilla, Johnston, Jean M., Acosta, Maria T., Nicoli, Elena-Raluca, Rothermel, Caroline, Thurm, Audrey, Karunakara, Ajith, Thorp, Benjamin, Ross, Peter, Jameson, John, Sheehan, Michael, Vaughn, Toby, Valencia, Donna, De Boever, Erika, Corcoran, Gavin
Format: Journal Article
Language:English
Published: Elsevier Inc 01-02-2022
Online Access:Get full text
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Description
ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2021.11.317