AAV-based gene therapy prevents neuropathology and results in normal cognitive development in the hyperargininemic mouse.
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Lee EK, et al. Among authors: rozengurt n.
Gene Ther. 2013 Aug;20(8):785-96. doi: 10.1038/gt.2012.99. Epub 2013 Feb 7.
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PMID: 23388701
Free PMC article.