AAV-based gene therapy prevents neuropathology and results in normal cognitive development in the hyperargininemic mouse
Lee EK, Hu C, Bhargava R, Ponnusamy R, Park H, Novicoff S, Rozengurt N, Marescau B, De Deyn P, Stout D, Schlichting L, Grody WW, Cederbaum SD, Lipshutz GS. AAV-based gene therapy prevents neuropathology and results in normal cognitive development in the hyperargininemic mouse. Gene Ther. 2013 Aug;20(8):785-96. doi: 10.1038/gt.2012.99. Epub 2013 Feb 7. PMID: 23388701; PMCID: PMC3679314.