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Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery | Synapse
May 14, 2026
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery
RA
Rebecca C. Ahrens-Nicklas
CK
Chelsea Kotch
Children's Hospital of Philadelphia
AR
Aoife M. Roche
California University of Pennsylvania
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Key Points
This research aims to investigate the occurrence of neuroepithelial tumors following the delivery of an AAV vector via the intracisternal route.
Conducted a randomized trial analyzing tumor incidence post AAV vector delivery
Monitored integration events in neural tissues
Assessed clinical outcomes and any adverse effects.
A higher incidence of neuroepithelial tumors observed in AAV group compared to control (p<0.05)
Integration sites identified predominantly in neural progenitor cells
Long-term follow-up indicates persistent tumor growth with associated neurological deficits.
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Ahrens-Nicklas et al. (Wed,) studied this question.
synapsesocial.com/papers/6a0593a24cfb5beb2ffb8ebe
https://doi.org/https://doi.org/10.1056/nejmoa2601608