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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. Passini MA, et al. Mol Ther. 2005 May;11(5):754-62. doi: 10.1016/j.ymthe.2005.01.011. Mol Ther. 2005. PMID: 15851014 Free article.
Niemann-Pick A disease (NPA) is a fatal lysosomal storage disorder caused by a deficiency in acid sphingomyelinase (ASM) activity. The lack of functional ASM results in cellular accumulation of sphingomyelin and cholesterol within distended lysosomes throughout the …
Niemann-Pick A disease (NPA) is a fatal lysosomal storage disorder caused by a deficiency in acid sphingomyelinase (ASM) activity. Th …
Comparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular delivery.
Treleaven CM, Tamsett T, Fidler JA, Taksir TV, Cheng SH, Shihabuddin LS, Dodge JC. Treleaven CM, et al. PLoS One. 2011 Jan 25;6(1):e16313. doi: 10.1371/journal.pone.0016313. PLoS One. 2011. PMID: 21283548 Free PMC article.
Niemann-Pick A (NPA) disease is a lysosomal storage disorder (LSD) caused by a deficiency in acid sphingomyelinase (ASM) activity. Previously, we reported that biochemical and functional abnormalities observed in ASM knockout (ASMKO) mice could be partially alleviat …
Niemann-Pick A (NPA) disease is a lysosomal storage disorder (LSD) caused by a deficiency in acid sphingomyelinase (ASM) activity. Pr …
Injection of mouse and human neural stem cells into neonatal Niemann-Pick A model mice.
Sidman RL, Li J, Stewart GR, Clarke J, Yang W, Snyder EY, Shihabuddin LS. Sidman RL, et al. Brain Res. 2007 Apr 6;1140:195-204. doi: 10.1016/j.brainres.2007.01.011. Epub 2007 Jan 9. Brain Res. 2007. PMID: 17289003
Cloned mouse C17.2 neural stem cells (NSCs) or human NSCs were injected into five CNS sites in very large numbers (100,000 cells/site, or a total of 500,000 cells) into 18 neonatal mice homozygous for a targeted deletion (knockout) of the acid sphingomyelinase (ASM) gene ( …
Cloned mouse C17.2 neural stem cells (NSCs) or human NSCs were injected into five CNS sites in very large numbers (100,000 cells/site, or a …
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. Yang WW, et al. Exp Neurol. 2007 Oct;207(2):258-66. doi: 10.1016/j.expneurol.2007.06.017. Epub 2007 Jul 10. Exp Neurol. 2007. PMID: 17686472
Niemann-Pick A disease (NPD-A) is caused by a deficiency of acid sphingomyelinase (ASM) leading to the intracellular accumulation of sphingomyelin and cholesterol in lysosomes. We evaluated the effects of direct intraparenchymal brain injections of purified recombinant hum …
Niemann-Pick A disease (NPD-A) is caused by a deficiency of acid sphingomyelinase (ASM) leading to the intracellular accumulation of …
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. Bu J, et al. Mol Ther. 2012 Oct;20(10):1893-901. doi: 10.1038/mt.2012.118. Epub 2012 Jul 24. Mol Ther. 2012. PMID: 22828503 Free PMC article.
Niemann-Pick disease Type A (NPA) is a neuronopathic lysosomal storage disease (LSD) caused by the loss of acid sphingomyelinase (ASM). The goals of the current study are to ascertain the levels of human ASM that are efficacious in ASM knockout (ASMKO) mice, …
Niemann-Pick disease Type A (NPA) is a neuronopathic lysosomal storage disease (LSD) caused by the loss of acid sphingomyelinase (ASM …
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. Salegio EA, et al. Hum Gene Ther. 2010 Sep;21(9):1093-103. doi: 10.1089/hum.2010.040. Hum Gene Ther. 2010. PMID: 20408734 Free PMC article.
Gene replacement therapy for the neurological deficits caused by lysosomal storage disorders, such as in Niemann-Pick disease type A, will require widespread expression of efficacious levels of acid sphingomyelinase (ASM) in the infant human brain. At present there is no t …
Gene replacement therapy for the neurological deficits caused by lysosomal storage disorders, such as in Niemann-Pick disease type A, will r …
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. Dodge JC, et al. Exp Neurol. 2009 Feb;215(2):349-57. doi: 10.1016/j.expneurol.2008.10.021. Epub 2008 Nov 14. Exp Neurol. 2009. PMID: 19059399
Niemann-Pick A (NPA) disease is a lysosomal storage disorder (LSD) caused by a deficiency in acid sphingomyelinase (ASM) activity. Previously, we showed that the storage pathology in the ASM knockout (ASMKO) mouse brain could be corrected by intracerebral injections …
Niemann-Pick A (NPA) disease is a lysosomal storage disorder (LSD) caused by a deficiency in acid sphingomyelinase (ASM) activity. Pr …
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. 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.
Niemann-Pick type A disease is a lysosomal storage disorder caused by a deficiency in acid sphingomyelinase (ASM) activity. Previously we showed that storage pathology in the ASM knockout (ASMKO) mouse brain can be corrected by adeno-associated virus serotype 2 (AAV …
Niemann-Pick type A disease is a lysosomal storage disorder caused by a deficiency in acid sphingomyelinase (ASM) activity. Previousl …
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. J Neurosci. 2004 Nov 24;24(47):10642-51. doi: 10.1523/JNEUROSCI.3584-04.2004. J Neurosci. 2004. PMID: 15564580 Free PMC article.
Niemann-Pick disease is caused by a genetic deficiency in acid sphingomyelinase (ASM) leading to the intracellular accumulation of sphingomyelin and cholesterol in lysosomes. ...NPCs derived from adult mouse brain were genetically modified to express human ASM (hASM …
Niemann-Pick disease is caused by a genetic deficiency in acid sphingomyelinase (ASM) leading to the intracellular accumulation of sp …
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. 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.
Niemann-Pick disease (NPD) is caused by the loss of acid sphingomyelinase (ASM) activity, which results in widespread accumulation of undegraded lipids in cells of the viscera and CNS. In this study, we tested the effect of combination brain and systemic injections of reco …
Niemann-Pick disease (NPD) is caused by the loss of acid sphingomyelinase (ASM) activity, which results in widespread accumulation of …