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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: yang ww. 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.
Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann-Pick-A mouse leads to a marked decrease in lysosomal storage pathology.
Shihabuddin LS, Numan S, Huff MR, Dodge JC, Clarke J, Macauley SL, Yang W, Taksir TV, Parsons G, Passini MA, Gage FH, Stewart GR. Shihabuddin LS, et al. Among authors: yang w. J Neurosci. 2004 Nov 24;24(47):10642-51. doi: 10.1523/JNEUROSCI.3584-04.2004. J Neurosci. 2004. PMID: 15564580 Free PMC article.
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: yang w. 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: yang w. J Neurosci. 2006 Feb 1;26(5):1334-42. doi: 10.1523/JNEUROSCI.2676-05.2006. J Neurosci. 2006. PMID: 16452657 Free PMC article.
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