Author

Sumaiya Ashraf

National Human Genome Research Institute

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· 2

Signature Assays, Organisms & Methods

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Approach:experimental assay· 2 Organism:human· 2 Research Focus:benchmarking· 1 Research Focus:drug response / drug discovery· 1

Frequent Co-Authors

Allison M. KeeleriD· 2 shared Amanda L. GrossiD· 2 shared Audrey ThurmiD· 2 shared Barry J. ByrneiD· 2 shared Connor J. LewisiD· 2 shared Cynthia J. TifftiD· 2 shared Douglas R. MartiniD· 2 shared Heather L. Gray‐EdwardsiD· 2 shared

Activity by Year

2026-2026 · peak 2/yr

Co-Author Collaboration Network

10 top collaborators · 45 mutual co-author links

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2 shared papersGenetic & Genomic Medicine

Allison M. KeelerORCID

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Author profile →

2 papers

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

AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial

Connor J. Lewis, Precilla D’Souza, Jean M. Johnston, Maria T. Acosta, Cristan Farmer, Eva H. Baker, Anna Crowell, Yoliann Mojica Algarin, Sumaiya Ashraf, Lisa A. Joseph, Gilbert Vézina, Zenaide Quezado, Muhammad H. Yousef, Zeynep Vardar, Mohammed Salman Shazeeb, , 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.

New England Journal of Medicine · Feb 6, 2026Peer-reviewedGenetic & Genomic Medicine