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Glucocerebrosidase 2 gene deletion rescues type 1 Gaucher disease.
Mistry PK, Liu J, Sun L, Chuang WL, Yuen T, Yang R, Lu P, Zhang K, Li J, Keutzer J, Stachnik A, Mennone A, Boyer JL, Jain D, Brady RO, New MI, Zaidi M. Mistry PK, et al. Proc Natl Acad Sci U S A. 2014 Apr 1;111(13):4934-9. doi: 10.1073/pnas.1400768111. Epub 2014 Mar 17. Proc Natl Acad Sci U S A. 2014. PMID: 24639522 Free PMC article.