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Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs.
Ponder KP, Melniczek JR, Xu L, Weil MA, O'Malley TM, O'Donnell PA, Knox VW, Aguirre GD, Mazrier H, Ellinwood NM, Sleeper M, Maguire AM, Volk SW, Mango RL, Zweigle J, Wolfe JH, Haskins ME. Ponder KP, et al. Proc Natl Acad Sci U S A. 2002 Oct 1;99(20):13102-7. doi: 10.1073/pnas.192353499. Epub 2002 Sep 13. Proc Natl Acad Sci U S A. 2002. PMID: 12232044 Free PMC article.