Author

Gilbert Vezina

Children's National

Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.

Biology Disciplines

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Genetic & Genomic Medicine· 1

Signature Assays, Organisms & Methods

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Approach:experimental assay· 1 Organism:human· 1

Frequent Co-Authors

Allison M. KeeleriD· 1 shared Amanda L. GrossiD· 1 shared Audrey ThurmiD· 1 shared Barry J. ByrneiD· 1 shared Connor J. LewisiD· 1 shared Cynthia J. TifftiD· 1 shared Douglas R. MartiniD· 1 shared Heather L. Gray‐EdwardsiD· 1 shared

Activity by Year

2026-2026 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 45 mutual co-author links

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1 shared paperGenetic & Genomic Medicine

Allison M. KeelerORCID

Mutual co-authors in cluster

Author profile →

1 paper

Recent·Most cited

Expanded Access use of Intravenous Gene Transfer with AAV9-GLB1 in a Juvenile GM1 Gangliosidosis Participant

Connor J. Lewis, Hera Akmal, Precilla D’Souza, Jean M. Johnston, Sumaiya Ashraf, Gilbert Vezina, Selby I. Chipman, Meghan Blackwood, Muhammad H. Yousef, Zenaide Quezado, William Allen Gahl, Barry J. Byrne, Terence R. Flotte, Xuntian Jiang, Audrey Thurm, , 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.

medRxiv · Sep 10, 2026 · PreprintGenetic & Genomic Medicine