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Prospects for the gene therapy of spinal muscular atrophy.
Passini MA, Cheng SH. Passini MA, et al. Among authors: cheng sh. Trends Mol Med. 2011 May;17(5):259-65. doi: 10.1016/j.molmed.2011.01.002. Epub 2011 Feb 19. Trends Mol Med. 2011. PMID: 21334976 Review.
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: cheng sh. 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: cheng sh. 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.
Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.
Passini MA, Dodge JC, Bu J, Yang W, Zhao Q, Sondhi D, Hackett NR, Kaminsky SM, Mao Q, Shihabuddin LS, Cheng SH, Sleat DE, Stewart GR, Davidson BL, Lobel P, Crystal RG. Passini MA, et al. Among authors: cheng sh. J Neurosci. 2006 Feb 1;26(5):1334-42. doi: 10.1523/JNEUROSCI.2676-05.2006. J Neurosci. 2006. PMID: 16452657 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: cheng sh. 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.
CNS expression of glucocerebrosidase corrects alpha-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy.
Sardi SP, Clarke J, Kinnecom C, Tamsett TJ, Li L, Stanek LM, Passini MA, Grabowski GA, Schlossmacher MG, Sidman RL, Cheng SH, Shihabuddin LS. Sardi SP, et al. Among authors: cheng sh. Proc Natl Acad Sci U S A. 2011 Jul 19;108(29):12101-6. doi: 10.1073/pnas.1108197108. Epub 2011 Jul 5. Proc Natl Acad Sci U S A. 2011. PMID: 21730160 Free PMC article.
984 results