01707nam a2200313   4500001000800000005001700008007000300025008004100028040000800069041000800077100003900085245009000124300002500214520077400239650001901013650002801032650002301060650001701083650002001100650001601120700002001136700002101156700002301177700002001200700002201220700002201242700002601264773010301290ESSALUD20260820162144.0ta      t        pe ||||| |||| 00| 0 spa d  aBMG  aeng  aAhrens-Nicklas, Rebecca C. eAutor  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 CEREBRAL  aENFERMEDADES INFANTILES  aGENÉTICA GENERAL  aNEUROLOGÍA  aNEUROCIRUGÍA   aPEDIATRÍA  aKotch, Chelsea   aRoche, Aoife M.   aEverett, John K.    aReddy, Shantan   aSanti, Mariarita   aMadsen, Peter J.   aMartos-Rus, Cristina 0 022717dMassachusetts NEJM GroupoNEJM011tThe New England Journal of Medicine wESSALUDx0028-4793