01870nam a2200337 4500 ESSALUD 20260820162144.0 ta t pe ||||| |||| 00| 0 spa d BMG eng Ahrens-Nicklas, Rebecca C. Autor 54204 Neuroepithelial tumor with AAV integration after intracisternal magna vector delivery páginas: 2126-2133 Recombinant adeno-associated virus (AAV) vectors are predominantly nonintegrating, but rare genomic integration events have been associated with oncogenesis in neonatal murine models. Here we report a case of a neuroepithelial tumor that developed in a 5-year-old boy with severe mucopolysaccharidosis type I (MPSI, Hurler subtype) 4 years after intracisternal magna administration of AAV serotype 9 gene therapy. The patient underwent successful resection of the primary tumor. Postoperatively, he has continued to have advanced cognitive function for his age, a finding that indicates mitigation of MPSI. Molecular analysis of the tumor showed clonal integration of rearranged AAV vector elements into the gene PLAG1 and expression of a chimeric AAV-PLAG1 transcript. TUMOR CEREBRAL 54205 ENFERMEDADES INFANTILES 53890 GENÉTICA GENERAL 54107 NEUROLOGÍA NEUROCIRUGÍA 7782 PEDIATRÍA 6401 Kotch, Chelsea 54206 Roche, Aoife M. 54207 Everett, John K. 54208 Reddy, Shantan 54209 Santi, Mariarita 54210 Madsen, Peter J. 54211 Martos-Rus, Cristina 54212 22717 22648 Massachusetts NEJM Group NEJM011 The New England Journal of Medicine ESSALUD 0028-4793 ARTICULOS 2026-08-20 sqb 22843 22843