01875nam a2200337   4500001000800000005001700008007000300025008004100028040000800069041000800077100004600085245009000131300002500221520077400246650002601020650003501046650003001081650001701111650002601128650002201154700002701176700002801203700003001231700002701261700002901288700002901317700003301346773011001379942003101489999001701520ESSALUD20260820162144.0ta      t        pe ||||| |||| 00| 0 spa d  aBMG  aeng  aAhrens-Nicklas, Rebecca C. eAutor954204  aNeuroepithelial tumor with AAV integration after intracisternal magna vector delivery  apáginas: 2126-2133  aRecombinant 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.  aTUMOR CEREBRAL954205  aENFERMEDADES INFANTILES953890  aGENÉTICA GENERAL954107  aNEUROLOGÍA  aNEUROCIRUGÍA 97782  aPEDIATRÍA96401  aKotch, Chelsea 954206  aRoche, Aoife M. 954207  aEverett, John K.  954208  aReddy, Shantan 954209  aSanti, Mariarita 954210  aMadsen, Peter J. 954211  aMartos-Rus, Cristina 9542120 022717922648dMassachusetts NEJM GroupoNEJM011tThe New England Journal of Medicine wESSALUDx0028-4793  cARTICULOSe2026-08-20zsqb  c22843d22843