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eP259: A phase 1/2 trial of AXO-AAV-GM1 gene therapy for the treatment of infantile- and juvenile-onset GM1 gangliosidosis
Genetics in Medicine, 2022 Cynthia Tift, Precilla D'Souza, Jean Johnston, Maria Acosta, Elena-Raluca Nicoli, Caroline Rothermel, Audrey Thurm, Ajith Karunakara, Benjamin Thorp, Peter Ross, John Jameson, Michael Sheehan, Toby Vaughn, Donna Valencia, Erika De Boever +14 moreopenaire +1 more source