Work

Expanded access use of intravenous gene transfer with AAV9 vector galactosidase in child with type II GM1 gangliosidosis

Year: 2026

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2025.109304

Authors: Hera Akmal, Precilla D'Souza, Jean M. Johnston, Connor J. Lewis, Maria T. Acosta, Cynthia J. Tifft

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

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: W7127423917

FWCI:

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