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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy.
Passini MA, Bu J, Richards AM, Treleaven CM, Sullivan JA, O'Riordan CR, Scaria A, Kells AP, Samaranch L, San Sebastian W, Federici T, Fiandaca MS, Boulis NM, Bankiewicz KS, Shihabuddin LS, Cheng SH. Passini MA, et al. Among authors: bu j. Hum Gene Ther. 2014 Jul;25(7):619-30. doi: 10.1089/hum.2014.011. Epub 2014 Apr 28. Hum Gene Ther. 2014. PMID: 24617515
Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease.
Dodge JC, Clarke J, Song A, Bu J, Yang W, Taksir TV, Griffiths D, Zhao MA, Schuchman EH, Cheng SH, O'Riordan CR, Shihabuddin LS, Passini MA, Stewart GR. Dodge JC, et al. Among authors: bu j. Proc Natl Acad Sci U S A. 2005 Dec 6;102(49):17822-7. doi: 10.1073/pnas.0509062102. Epub 2005 Nov 21. Proc Natl Acad Sci U S A. 2005. PMID: 16301517 Free PMC article.
Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse.
Passini MA, Bu J, Fidler JA, Ziegler RJ, Foley JW, Dodge JC, Yang WW, Clarke J, Taksir TV, Griffiths DA, Zhao MA, O'Riordan CR, Schuchman EH, Shihabuddin LS, Cheng SH. Passini MA, et al. Among authors: bu j. Proc Natl Acad Sci U S A. 2007 May 29;104(22):9505-10. doi: 10.1073/pnas.0703509104. Epub 2007 May 21. Proc Natl Acad Sci U S A. 2007. PMID: 17517638 Free PMC article.
Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease.
Cabrera-Salazar MA, Roskelley EM, Bu J, Hodges BL, Yew N, Dodge JC, Shihabuddin LS, Sohar I, Sleat DE, Scheule RK, Davidson BL, Cheng SH, Lobel P, Passini MA. Cabrera-Salazar MA, et al. Among authors: bu j. Mol Ther. 2007 Oct;15(10):1782-8. doi: 10.1038/sj.mt.6300249. Epub 2007 Jul 17. Mol Ther. 2007. PMID: 17637720 Free article.
785 results