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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. Among authors: brady ro. 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.
Gaucher disease: resetting the clinical and scientific agenda.
Mistry PK, Weinreb NJ, Brady RO, Grabowski GA. Mistry PK, et al. Among authors: brady ro. Am J Hematol. 2009 Apr;84(4):205-7. doi: 10.1002/ajh.21384. Am J Hematol. 2009. PMID: 19296473 Free PMC article. No abstract available.
Effective cell and gene therapy in a murine model of Gaucher disease.
Enquist IB, Nilsson E, Ooka A, Månsson JE, Olsson K, Ehinger M, Brady RO, Richter J, Karlsson S. Enquist IB, et al. Among authors: brady ro. Proc Natl Acad Sci U S A. 2006 Sep 12;103(37):13819-24. doi: 10.1073/pnas.0606016103. Epub 2006 Sep 5. Proc Natl Acad Sci U S A. 2006. PMID: 16954197 Free PMC article.
Decreased glucocerebrosidase activity in Gaucher disease parallels quantitative enzyme loss due to abnormal interaction with TCP1 and c-Cbl.
Lu J, Chiang J, Iyer RR, Thompson E, Kaneski CR, Xu DS, Yang C, Chen M, Hodes RJ, Lonser RR, Brady RO, Zhuang Z. Lu J, et al. Among authors: brady ro. Proc Natl Acad Sci U S A. 2010 Dec 14;107(50):21665-70. doi: 10.1073/pnas.1014376107. Epub 2010 Nov 22. Proc Natl Acad Sci U S A. 2010. PMID: 21098288 Free PMC article.
Murine models of acute neuronopathic Gaucher disease.
Enquist IB, Lo Bianco C, Ooka A, Nilsson E, Månsson JE, Ehinger M, Richter J, Brady RO, Kirik D, Karlsson S. Enquist IB, et al. Among authors: brady ro. Proc Natl Acad Sci U S A. 2007 Oct 30;104(44):17483-8. doi: 10.1073/pnas.0708086104. Epub 2007 Oct 22. Proc Natl Acad Sci U S A. 2007. PMID: 17954912 Free PMC article.
538 results