eP259: A phase 1/2 trial of AXO-AAV-GM1 gene therapy for the treatment of infantile- and juvenile-onset GM1 gangliosidosis

• DOI: 10.1016/j.gim.2022.01.294
• Publication Date: 2022-03-21T09:30:37Z
• Authors: 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
• Keywords: 0303 health sciences; 03 medical and health sciences