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Items: 46


SMN regulates axonal local translation via miR-183/mTOR pathway.

Kye MJ, Niederst ED, Wertz MH, Gonçalves Ido C, Akten B, Dover KZ, Peters M, Riessland M, Neveu P, Wirth B, Kosik KS, Sardi SP, Monani UR, Passini MA, Sahin M.

Hum Mol Genet. 2014 Dec 1;23(23):6318-31. doi: 10.1093/hmg/ddu350. Epub 2014 Jul 4.


Systemic administration of a recombinant AAV1 vector encoding IGF-1 improves disease manifestations in SMA mice.

Tsai LK, Chen CL, Ting CH, Lin-Chao S, Hwu WL, Dodge JC, Passini MA, Cheng SH.

Mol Ther. 2014 Aug;22(8):1450-1459. doi: 10.1038/mt.2014.84. Epub 2014 May 12.


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.

Hum Gene Ther. 2014 Jul;25(7):619-30. doi: 10.1089/hum.2014.011. Epub 2014 Apr 28.


Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies.

Sardi SP, Clarke J, Viel C, Chan M, Tamsett TJ, Treleaven CM, Bu J, Sweet L, Passini MA, Dodge JC, Yu WH, Sidman RL, Cheng SH, Shihabuddin LS.

Proc Natl Acad Sci U S A. 2013 Feb 26;110(9):3537-42. doi: 10.1073/pnas.1220464110. Epub 2013 Jan 7.


Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A.

Bu J, Ashe KM, Bringas J, Marshall J, Dodge JC, Cabrera-Salazar MA, Forsayeth J, Schuchman EH, Bankiewicz KS, Cheng SH, Shihabuddin LS, Passini MA.

Mol Ther. 2012 Oct;20(10):1893-901. doi: 10.1038/mt.2012.118. Epub 2012 Jul 24.


Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies.

Treleaven CM, Tamsett TJ, Bu J, Fidler JA, Sardi SP, Hurlbut GD, Woodworth LA, Cheng SH, Passini MA, Shihabuddin LS, Dodge JC.

Mol Ther. 2012 Sep;20(9):1713-23. doi: 10.1038/mt.2012.114. Epub 2012 Jun 26.


Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs.

Federici T, Taub JS, Baum GR, Gray SJ, Grieger JC, Matthews KA, Handy CR, Passini MA, Samulski RJ, Boulis NM.

Gene Ther. 2012 Aug;19(8):852-9. doi: 10.1038/gt.2011.130. Epub 2011 Sep 15.


IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice.

Tsai LK, Chen YC, Cheng WC, Ting CH, Dodge JC, Hwu WL, Cheng SH, Passini MA.

Neurobiol Dis. 2012 Jan;45(1):272-9. doi: 10.1016/j.nbd.2011.06.021. Epub 2011 Aug 18.


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.

Proc Natl Acad Sci U S A. 2011 Jul 19;108(29):12101-6. doi: 10.1073/pnas.1108197108. Epub 2011 Jul 5.


Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.

Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, Stanek LM, Hua Y, Rigo F, Matson J, Hung G, Kaye EM, Shihabuddin LS, Krainer AR, Bennett CF, Cheng SH.

Sci Transl Med. 2011 Mar 2;3(72):72ra18. doi: 10.1126/scitranslmed.3001777.


Prospects for the gene therapy of spinal muscular atrophy.

Passini MA, Cheng SH.

Trends Mol Med. 2011 May;17(5):259-65. doi: 10.1016/j.molmed.2011.01.002. Epub 2011 Feb 19. Review.


Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR.

Genes Dev. 2010 Aug 1;24(15):1634-44. doi: 10.1101/gad.1941310. Epub 2010 Jul 12.


Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders.

Salegio EA, Kells AP, Richardson RM, Hadaczek P, Forsayeth J, Bringas J, Sardi SP, Passini MA, Shihabuddin LS, Cheng SH, Fiandaca MS, Bankiewicz KS.

Hum Gene Ther. 2010 Sep;21(9):1093-103. doi: 10.1089/hum.2010.040.


CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.

Passini MA, Bu J, Roskelley EM, Richards AM, Sardi SP, O'Riordan CR, Klinger KW, Shihabuddin LS, Cheng SH.

J Clin Invest. 2010 Apr;120(4):1253-64. doi: 10.1172/JCI41615. Epub 2010 Mar 15.


Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters.

Husain T, Passini MA, Parente MK, Fraser NW, Wolfe JH.

Gene Ther. 2009 Jul;16(7):927-32. doi: 10.1038/gt.2009.52. Epub 2009 May 21.


Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease.

Dodge JC, Clarke J, Treleaven CM, Taksir TV, Griffiths DA, Yang W, Fidler JA, Passini MA, Karey KP, Schuchman EH, Cheng SH, Shihabuddin LS.

Exp Neurol. 2009 Feb;215(2):349-57. doi: 10.1016/j.expneurol.2008.10.021. Epub 2008 Nov 14.


Development of a single vector system that enhances trans-splicing of SMN2 transcripts.

Coady TH, Baughan TD, Shababi M, Passini MA, Lorson CL.

PLoS One. 2008;3(10):e3468. doi: 10.1371/journal.pone.0003468. Epub 2008 Oct 22.


Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice.

Sidman RL, Taksir T, Fidler J, Zhao M, Dodge JC, Passini MA, Raben N, Thurberg BL, Cheng SH, Shihabuddin LS.

J Neuropathol Exp Neurol. 2008 Aug;67(8):803-18. doi: 10.1097/NEN.0b013e3181815994.


Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity.

Dodge JC, Haidet AM, Yang W, Passini MA, Hester M, Clarke J, Roskelley EM, Treleaven CM, Rizo L, Martin H, Kim SH, Kaspar R, Taksir TV, Griffiths DA, Cheng SH, Shihabuddin LS, Kaspar BK.

Mol Ther. 2008 Jun;16(6):1056-64. doi: 10.1038/mt.2008.60. Epub 2008 Apr 1.


Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis.

Chang M, Cooper JD, Sleat DE, Cheng SH, Dodge JC, Passini MA, Lobel P, Davidson BL.

Mol Ther. 2008 Apr;16(4):649-56. doi: 10.1038/mt.2008.9. Epub 2008 Feb 12.


Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouse.

Yang WW, Dodge JC, Passini MA, Taksir TV, Griffiths D, Schuchman EH, Cheng SH, Shihabuddin LS.

Exp Neurol. 2007 Oct;207(2):258-66. Epub 2007 Jul 10.


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.

Mol Ther. 2007 Oct;15(10):1782-8. Epub 2007 Jul 17.


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.

Proc Natl Acad Sci U S A. 2007 May 29;104(22):9505-10. Epub 2007 May 21.


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.

J Neurosci. 2006 Feb 1;26(5):1334-42.


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.

Proc Natl Acad Sci U S A. 2005 Dec 6;102(49):17822-7. Epub 2005 Nov 21.


Egr3 stimulation of GABRA4 promoter activity as a mechanism for seizure-induced up-regulation of GABA(A) receptor alpha4 subunit expression.

Roberts DS, Raol YH, Bandyopadhyay S, Lund IV, Budreck EC, Passini MA, Wolfe JH, Brooks-Kayal AR, Russek SJ.

Proc Natl Acad Sci U S A. 2005 Aug 16;102(33):11894-9. Epub 2005 Aug 9. Erratum in: Proc Natl Acad Sci U S A. 2005 Sep 13;102(37):13351. Passini, MJ [corrected to Passini, MA].


AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease.

Passini MA, Macauley SL, Huff MR, Taksir TV, Bu J, Wu IH, Piepenhagen PA, Dodge JC, Shihabuddin LS, O'Riordan CR, Schuchman EH, Stewart GR.

Mol Ther. 2005 May;11(5):754-62.


AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy.

Rafi MA, Zhi Rao H, Passini MA, Curtis M, Vanier MT, Zaka M, Luzi P, Wolfe JH, Wenger DA.

Mol Ther. 2005 May;11(5):734-44.


Effective gene therapy for an inherited CNS disease in a large animal model.

Vite CH, McGowan JC, Niogi SN, Passini MA, Drobatz KJ, Haskins ME, Wolfe JH.

Ann Neurol. 2005 Mar;57(3):355-64.


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.

J Neurosci. 2004 Nov 24;24(47):10642-51.


A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration.

Sleat DE, Wiseman JA, El-Banna M, Kim KH, Mao Q, Price S, Macauley SL, Sidman RL, Shen MM, Zhao Q, Passini MA, Davidson BL, Stewart GR, Lobel P.

J Neurosci. 2004 Oct 13;24(41):9117-26.


Gene delivery to the mouse brain with adeno-associated virus.

Passini MA, Watson DJ, Wolfe JH.

Methods Mol Biol. 2004;246:225-36.


Adeno-associated virus vector-mediated transduction in the cat brain.

Vite CH, Passini MA, Haskins ME, Wolfe JH.

Gene Ther. 2003 Oct;10(22):1874-81.


Genetically modified NT2N human neuronal cells mediate long-term gene expression as CNS grafts in vivo and improve functional cognitive outcome following experimental traumatic brain injury.

Watson DJ, Longhi L, Lee EB, Fulp CT, Fujimoto S, Royo NC, Passini MA, Trojanowski JQ, Lee VM, McIntosh TK, Wolfe JH.

J Neuropathol Exp Neurol. 2003 Apr;62(4):368-80.


Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible.

Heuer GG, Passini MA, Jiang K, Parente MK, Lee VM, Trojanowski JQ, Wolfe JH.

Ann Neurol. 2002 Dec;52(6):762-70.


Cloning of zebrafish vsx1: expression of a paired-like homeobox gene during CNS development.

Passini MA, Kurtzman AL, Canger AK, Asch WS, Wray GA, Raymond PA, Schechter N.

Dev Genet. 1998;23(2):128-41.


Restricted expression of the neuronal intermediate filament protein plasticin during zebrafish development.

Canger AK, Passini MA, Asch WS, Leake D, Zafonte BT, Glasgow E, Schechter N.

J Comp Neurol. 1998 Oct 5;399(4):561-72.


Vsx-2, a gene encoding a paired-type homeodomain, is expressed in the retina, hindbrain, and spinal cord during goldfish embryogenesis.

Passini MA, Raymond PA, Schechter N.

Brain Res Dev Brain Res. 1998 Aug 8;109(2):129-35.


Cloning of zebrafish neurofilament cDNAs for plasticin and gefiltin: increased mRNA expression in ganglion cells after optic nerve injury.

Asch WS, Leake D, Canger AK, Passini MA, Argenton F, Schechter N.

J Neurochem. 1998 Jul;71(1):20-32.


Vsx-1 and Vsx-2: differential expression of two paired-like homeobox genes during zebrafish and goldfish retinogenesis.

Passini MA, Levine EM, Canger AK, Raymond PA, Schechter N.

J Comp Neurol. 1997 Nov 24;388(3):495-505.


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