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Effective gene therapy in an authentic model of Tay-Sachs-related diseases.
Cachón-González MB, Wang SZ, Lynch A, Ziegler R, Cheng SH, Cox TM. Cachón-González MB, et al. Among authors: cox tm. Proc Natl Acad Sci U S A. 2006 Jul 5;103(27):10373-10378. doi: 10.1073/pnas.0603765103. Epub 2006 Jun 26. Proc Natl Acad Sci U S A. 2006. PMID: 16801539 Free PMC article.
Insights into Krabbe disease from structures of galactocerebrosidase.
Deane JE, Graham SC, Kim NN, Stein PE, McNair R, Cachón-González MB, Cox TM, Read RJ. Deane JE, et al. Among authors: cox tm. Proc Natl Acad Sci U S A. 2011 Sep 13;108(37):15169-73. doi: 10.1073/pnas.1105639108. Epub 2011 Aug 29. Proc Natl Acad Sci U S A. 2011. PMID: 21876145 Free PMC article.
Characterization of inducible models of Tay-Sachs and related disease.
Sargeant TJ, Drage DJ, Wang S, Apostolakis AA, Cox TM, Cachón-González MB. Sargeant TJ, et al. Among authors: cox tm. PLoS Genet. 2012 Sep;8(9):e1002943. doi: 10.1371/journal.pgen.1002943. Epub 2012 Sep 20. PLoS Genet. 2012. PMID: 23028353 Free PMC article.
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
Bradbury AM, Cochran JN, McCurdy VJ, Johnson AK, Brunson BL, Gray-Edwards H, Leroy SG, Hwang M, Randle AN, Jackson LS, Morrison NE, Baek RC, Seyfried TN, Cheng SH, Cox NR, Baker HJ, Cachón-González MB, Cox TM, Sena-Esteves M, Martin DR. Bradbury AM, et al. Among authors: cox nr, cox tm. Mol Ther. 2013 Jul;21(7):1306-15. doi: 10.1038/mt.2013.86. Epub 2013 May 21. Mol Ther. 2013. PMID: 23689599 Free PMC article.
Reversibility of neuropathology in Tay-Sachs-related diseases.
Cachón-González MB, Wang SZ, Ziegler R, Cheng SH, Cox TM. Cachón-González MB, et al. Among authors: cox tm. Hum Mol Genet. 2014 Feb 1;23(3):730-48. doi: 10.1093/hmg/ddt459. Epub 2013 Sep 20. Hum Mol Genet. 2014. PMID: 24057669 Free PMC article.
276 results