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Wyświetlanie 1-20 z 20
Tytuł :
Neutral Lipid Cacostasis Contributes to Disease Pathogenesis in Amyotrophic Lateral Sclerosis.
Autorzy :
Dodge JC; Rare and Neurological Diseases Therapeutic Area .
Jensen EH; Rare and Neurological Diseases Therapeutic Area.
Yu J; Rare and Neurological Diseases Therapeutic Area.
Sardi SP; Rare and Neurological Diseases Therapeutic Area.
Bialas AR; Rare and Neurological Diseases Therapeutic Area.
Taksir TV; Global Discovery Pathology, Sanofi R+D, Framingham, Massachusetts 01701.
Bangari DS; Global Discovery Pathology, Sanofi R+D, Framingham, Massachusetts 01701.
Shihabuddin LS; Rare and Neurological Diseases Therapeutic Area.
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Źródło :
The Journal of neuroscience : the official journal of the Society for Neuroscience [J Neurosci] 2020 Nov 18; Vol. 40 (47), pp. 9137-9147. Date of Electronic Publication: 2020 Oct 13.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis.
Autorzy :
Dodge JC; Genzyme, Framingham, MA 01701-9322, USA. />Treleaven CM
Fidler JA
Tamsett TJ
Bao C
Searles M
Taksir TV
Misra K
Sidman RL
Cheng SH
Shihabuddin LS
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Źródło :
Proceedings of the National Academy of Sciences of the United States of America [Proc Natl Acad Sci U S A] 2013 Jun 25; Vol. 110 (26), pp. 10812-7. Date of Electronic Publication: 2013 Jun 10.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural
MeSH Terms :
Amyotrophic Lateral Sclerosis/*metabolism
Acidosis/etiology ; Acidosis/genetics ; Acidosis/metabolism ; Amyotrophic Lateral Sclerosis/etiology ; Amyotrophic Lateral Sclerosis/genetics ; Animals ; Disease Models, Animal ; Disease Progression ; Glycogen/metabolism ; Humans ; Mice ; Mice, Transgenic ; Mutation ; Superoxide Dismutase/genetics ; Superoxide Dismutase/metabolism
Czasopismo naukowe
Tytuł :
Optimization of a histopathological biomarker for sphingomyelin accumulation in acid sphingomyelinase deficiency.
Autorzy :
Taksir TV; Department of Pathology, Genzyme, a Sanofi Company, Framingham, MA 01701, USA. tatyana.taksir@genzyme.com
Johnson J
Maloney CL
Yandl E
Griffiths D
Thurberg BL
Ryan S
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Źródło :
The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society [J Histochem Cytochem] 2012 Aug; Vol. 60 (8), pp. 620-9. Date of Electronic Publication: 2012 May 21.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Histocytological Preparation Techniques/*methods
Niemann-Pick Diseases/*metabolism
Sphingomyelins/*metabolism
Animals ; Biomarkers/metabolism ; Borates ; Epoxy Resins ; Humans ; Indicators and Reagents ; Liver/metabolism ; Mice ; Mice, Knockout ; Niemann-Pick Diseases/pathology ; Organ Specificity ; Phthalic Anhydrides ; Staining and Labeling ; Tannins ; Tissue Embedding ; Tolonium Chloride ; Toxins, Biological
Czasopismo naukowe
Tytuł :
Relationship between neuropathology and disease progression in the SOD1(G93A) ALS mouse.
Autorzy :
Yang WW; Genzyme Corporation, 49 New York Ave, Framingham, MA 01701-9322, USA. />Sidman RL
Taksir TV
Treleaven CM
Fidler JA
Cheng SH
Dodge JC
Shihabuddin LS
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Źródło :
Experimental neurology [Exp Neurol] 2011 Feb; Vol. 227 (2), pp. 287-95. Date of Electronic Publication: 2010 Dec 09.
Typ publikacji :
Comparative Study; Journal Article
MeSH Terms :
Disease Progression*
Amyotrophic Lateral Sclerosis/*enzymology
Amyotrophic Lateral Sclerosis/*pathology
Superoxide Dismutase/*biosynthesis
Alanine/genetics ; Amino Acid Substitution/genetics ; Amyotrophic Lateral Sclerosis/genetics ; Animals ; Disease Models, Animal ; Female ; Glycine/genetics ; Humans ; Male ; Mice ; Mice, Transgenic ; Superoxide Dismutase/genetics
Czasopismo naukowe
Tytuł :
Comparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular delivery.
Autorzy :
Treleaven CM; Genzyme Corporation, Framingham, Massachusetts, United States of America.
Tamsett T
Fidler JA
Taksir TV
Cheng SH
Shihabuddin LS
Dodge JC
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Źródło :
PloS one [PLoS One] 2011 Jan 25; Vol. 6 (1), pp. e16313. Date of Electronic Publication: 2011 Jan 25.
Typ publikacji :
Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Central Nervous System/*enzymology
Sphingomyelin Phosphodiesterase/*metabolism
Animals ; Dose-Response Relationship, Drug ; Infusions, Intraventricular ; Mice ; Organ Specificity ; Rats
Czasopismo naukowe
Tytuł :
AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice.
Autorzy :
Dodge JC; Genzyme Corporation, Framingham, Massachusetts 01701-9322, USA. />Treleaven CM
Fidler JA
Hester M
Haidet A
Handy C
Rao M
Eagle A
Matthews JC
Taksir TV
Cheng SH
Shihabuddin LS
Kaspar BK
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Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2010 Dec; Vol. 18 (12), pp. 2075-84. Date of Electronic Publication: 2010 Sep 21.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural
MeSH Terms :
Genetic Therapy*
Amyotrophic Lateral Sclerosis/*therapy
Insulin-Like Growth Factor I/*metabolism
Vascular Endothelial Growth Factor A/*metabolism
Amyotrophic Lateral Sclerosis/genetics ; Amyotrophic Lateral Sclerosis/metabolism ; Animals ; Central Nervous System/metabolism ; Dependovirus/genetics ; Disease Models, Animal ; Disease-Free Survival ; Embryonic Stem Cells ; Female ; Immunohistochemistry ; Insulin-Like Growth Factor I/genetics ; Male ; Mice ; Mice, Transgenic ; Reverse Transcriptase Polymerase Chain Reaction ; Vascular Endothelial Growth Factor A/genetics
Czasopismo naukowe
Tytuł :
Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease.
Autorzy :
Dodge JC; Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701, USA. />Clarke J
Treleaven CM
Taksir TV
Griffiths DA
Yang W
Fidler JA
Passini MA
Karey KP
Schuchman EH
Cheng SH
Shihabuddin LS
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Źródło :
Experimental neurology [Exp Neurol] 2009 Feb; Vol. 215 (2), pp. 349-57. Date of Electronic Publication: 2008 Nov 14.
Typ publikacji :
Journal Article
MeSH Terms :
Niemann-Pick Disease, Type A/*drug therapy
Sphingomyelin Phosphodiesterase/*administration & dosage
Animals ; Brain/metabolism ; Cholesterol/metabolism ; Disease Models, Animal ; Humans ; Injections, Intraventricular/methods ; Lysosomes/drug effects ; Lysosomes/metabolism ; Mice ; Mice, Knockout ; Niemann-Pick Disease, Type A/genetics ; Niemann-Pick Disease, Type A/pathology ; Sphingomyelin Phosphodiesterase/deficiency ; Sphingomyelin Phosphodiesterase/metabolism ; Sphingomyelins/metabolism ; Time Factors
Czasopismo naukowe
Tytuł :
Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity.
Autorzy :
Dodge JC; Genzyme Corporation, Framingham, Massachusetts 01701-9322, USA. />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
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Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2008 Jun; Vol. 16 (6), pp. 1056-64. Date of Electronic Publication: 2008 Apr 01.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Amyotrophic Lateral Sclerosis/*genetics
Amyotrophic Lateral Sclerosis/*therapy
Central Nervous System/*cytology
Dependovirus/*genetics
Genetic Therapy/*methods
Insulin-Like Growth Factor I/*genetics
Neuroglia/*cytology
Neuroglia/*metabolism
Animals ; Cell Survival ; Central Nervous System/metabolism ; Cerebellum/metabolism ; Female ; Insulin-Like Growth Factor I/metabolism ; Male ; Mice ; Neurodegenerative Diseases/metabolism ; Tumor Necrosis Factor-alpha/metabolism
Czasopismo naukowe
Tytuł :
Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice.
Autorzy :
Ziegler RJ; Genzyme, Framingham, MA 01701, USA.
Bercury SD
Fidler J
Zhao MA
Foley J
Taksir TV
Ryan S
Hodges BL
Scheule RK
Shihabuddin LS
Cheng SH
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Źródło :
Human gene therapy [Hum Gene Ther] 2008 Jun; Vol. 19 (6), pp. 609-21.
Typ publikacji :
Journal Article
MeSH Terms :
Dependovirus*
Genetic Vectors*
Glycogen Storage Disease Type II/*physiopathology
Glycogen Storage Disease Type II/*therapy
Liver/*enzymology
alpha-Glucosidases/*genetics
Animals ; Disease Models, Animal ; Glycogen Storage Disease Type II/complications ; Humans ; Liver Glycogen/genetics ; Liver Glycogen/metabolism ; Mice ; Mice, Mutant Strains ; Motor Activity ; Muscle, Skeletal/pathology ; Muscle, Skeletal/physiopathology ; Muscular Diseases/etiology ; Muscular Diseases/physiopathology ; Muscular Diseases/therapy ; alpha-Glucosidases/blood
Czasopismo naukowe
Tytuł :
Optimized preservation of CNS morphology for the identification of glycogen in the Pompe mouse model.
Autorzy :
Taksir TV; Department of Pathology, Genzyme Corporation, One Mountain Road, Framingham, MA 01701-9322, USA. tatyana.taksir@genzyme.com
Griffiths D
Johnson J
Ryan S
Shihabuddin LS
Thurberg BL
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Źródło :
The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society [J Histochem Cytochem] 2007 Oct; Vol. 55 (10), pp. 991-8. Date of Electronic Publication: 2007 May 17.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Tissue Fixation*
Brain/*pathology
Glycogen/*metabolism
Glycogen Storage Disease Type II/*pathology
Animals ; Brain/metabolism ; Buffers ; Cerebellum/pathology ; Disease Models, Animal ; Fixatives ; Formaldehyde ; Mice ; Mice, Inbred C57BL ; Mice, Knockout ; Paraffin Embedding ; Periodic Acid
Czasopismo naukowe
Tytuł :
Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann-Pick A mouse.
Autorzy :
Yang WW; Genzyme Corporation, One Mountain Road, Framingham, MA 01701-9322, USA.
Dodge JC
Passini MA
Taksir TV
Griffiths D
Schuchman EH
Cheng SH
Shihabuddin LS
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Źródło :
Experimental neurology [Exp Neurol] 2007 Oct; Vol. 207 (2), pp. 258-66. Date of Electronic Publication: 2007 Jul 10.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural
MeSH Terms :
Lysosomes/*metabolism
Niemann-Pick Disease, Type A/*drug therapy
Niemann-Pick Disease, Type A/*pathology
Sphingomyelin Phosphodiesterase/*therapeutic use
Animals ; Disease Models, Animal ; Dose-Response Relationship, Drug ; Drug Administration Routes ; Filipin/metabolism ; Mice ; Mice, Knockout ; Niemann-Pick Disease, Type A/genetics ; Sphingomyelin Phosphodiesterase/deficiency ; Time Factors ; Toxins, Biological/metabolism
Czasopismo naukowe
Tytuł :
Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse.
Autorzy :
Passini MA; Genzyme Corporation, Framingham, MA 01701, USA. />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
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Źródło :
Proceedings of the National Academy of Sciences of the United States of America [Proc Natl Acad Sci U S A] 2007 May 29; Vol. 104 (22), pp. 9505-10. Date of Electronic Publication: 2007 May 21.
Typ publikacji :
Journal Article
MeSH Terms :
Brain/*enzymology
Brain/*pathology
Dependovirus/*genetics
Niemann-Pick Diseases/*genetics
Niemann-Pick Diseases/*therapy
Animals ; Gene Expression Regulation, Enzymologic ; Genetic Therapy ; Genetic Vectors/genetics ; Humans ; Mice ; Mice, Knockout ; Niemann-Pick Diseases/enzymology ; Niemann-Pick Diseases/pathology ; Sphingomyelin Phosphodiesterase/deficiency ; Sphingomyelin Phosphodiesterase/genetics ; Sphingomyelin Phosphodiesterase/metabolism ; Sphingomyelins/metabolism ; Survival Rate
Czasopismo naukowe
    Wyświetlanie 1-20 z 20

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