Work

Clinical, neurological, and neuroradiological outcomes of intravenous AAV9 gene therapy in infantile GM1 gangliosidosis patients

Year: 2025

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2024.108975

Authors: Andrew J. Wang, Connor J. Lewis, Jean M. Johnston, Precilla D'Souza, Gilbert Vezina, Maria T. Acosta, Cynthia J. Tifft

Institutions: National Institutes of Health, National Human Genome Research Institute, Children's National

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology (medical)

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4406972452

FWCI:

Cites:

Cited by:

Related to: 10