Work

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

Year: 2022

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2021.11.317

Authors: Cynthia Tifft, Precilla D’Souza, Jean M. Johnston, Maria T. 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, Gavin Corcoran

Institutions: National Human Genome Research Institute, National Institute of Mental Health

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4210769662

FWCI:

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