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001			ESSALUD
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040			_aBMG
041			_aeng
100			_aAhrens-Nicklas, Rebecca C. _eAutor _954204
245			_aNeuroepithelial tumor with AAV integration after intracisternal magna vector delivery
300			_apáginas: 2126-2133
520			_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.
650			_aTUMOR CEREBRAL _954205
650			_aENFERMEDADES INFANTILES _953890
650			_aGENÉTICA GENERAL _954107
650			_aNEUROLOGÍA
650			_aNEUROCIRUGÍA _97782
650			_aPEDIATRÍA _96401
700			_aKotch, Chelsea _954206
700			_aRoche, Aoife M. _954207
700			_aEverett, John K. _954208
700			_aReddy, Shantan _954209
700			_aSanti, Mariarita _954210
700			_aMadsen, Peter J. _954211
700			_aMartos-Rus, Cristina _954212
773	0		_022717 _922648 _dMassachusetts NEJM Group _oNEJM011 _tThe New England Journal of Medicine _wESSALUD _x0028-4793
942			_cARTICULOS _e2026-08-20 _zsqb
999			_c22843 _d22843