Genetic & Genomic MedicineNew England Journal of MedicinePeer-reviewed
AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial
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.
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