Author
Washington University in St. LouisORCID 0000-0001-9048-7294Unclaimed ORCID profile · follow to get notified of new preprints
Mutual co-authors in cluster
4 papers
Recent·Most cited
Connor J. Lewis, Hera Akmal, Precilla D’Souza…·Cynthia J. Tifft
This study reports 5-year safety and 3-year clinical outcomes of AAV9-GLB1 gene therapy in a juvenile GM1 gangliosidosis patient, showing sustained seizure freedom and biochemical improvements.
Rodrigo T. Starosta, Hannah Saeger, Johanna ten Hoeve…·Neal K. Bennett
The paper identifies metabolic deficits in a Krabbe disease model, including enhanced ceramide synthesis and disrupted glycosylation, suggesting new therapeutic targets.
Connor J. Lewis, Precilla D’Souza, Jean M. Johnston…·Cynthia J. Tifft
This phase 1/2 trial evaluates AAV9 gene therapy for GM1 gangliosidosis Type II, showing safety and some biochemical and neuroimaging improvements.
Chase Chen, Charis Ma, Richard Sam…·Yu Chen
The paper investigates how GBA1 mutations affect glycoprotein nonmetastatic melanoma protein B (GPNMB) expression in dopaminergic neurons, linking lipid accumulation to Parkinson's disease risk.