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| Title : Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis - Hu_2012_Mol.Genet.Metab_107_213 |
| Author(s) : Hu J , Lu JY , Wong AM , Hynan LS , Birnbaum SG , Yilmaz DS , Streit BM , Lenartowicz EM , Thompson TC , Cooper JD , Hofmann SL |
| Ref : Mol Genet Metab , 107 :213 , 2012 |
| Abstract : Hu_2012_Mol.Genet.Metab_107_213 |
| ESTHER : Hu_2012_Mol.Genet.Metab_107_213 |
| PubMedSearch : Hu_2012_Mol.Genet.Metab_107_213 |
| PubMedID: 22704978 |
| Gene_locus related to this paper: mouse-ppt |
| Title : Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis - Macauley_2012_Ann.Neurol_71_797 |
| Author(s) : Macauley SL , Roberts MS , Wong AM , McSloy F , Reddy AS , Cooper JD , Sands MS |
| Ref : Annals of Neurology , 71 :797 , 2012 |
| Abstract : Macauley_2012_Ann.Neurol_71_797 |
| ESTHER : Macauley_2012_Ann.Neurol_71_797 |
| PubMedSearch : Macauley_2012_Ann.Neurol_71_797 |
| PubMedID: 22368049 |
| Gene_locus related to this paper: mouse-ppt |
| Title : Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis - Roberts_2012_J.Inherit.Metab.Dis_35_847 |
| Author(s) : Roberts MS , Macauley SL , Wong AM , Yilmas D , Hohm S , Cooper JD , Sands MS |
| Ref : J Inherit Metab Dis , 35 :847 , 2012 |
| Abstract : Roberts_2012_J.Inherit.Metab.Dis_35_847 |
| ESTHER : Roberts_2012_J.Inherit.Metab.Dis_35_847 |
| PubMedSearch : Roberts_2012_J.Inherit.Metab.Dis_35_847 |
| PubMedID: 22310926 |
| Gene_locus related to this paper: mouse-ppt |
| Title : Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease - Kielar_2009_Hum.Mol.Genet_18_4066 |
| Author(s) : Kielar C , Wishart TM , Palmer A , Dihanich S , Wong AM , Macauley SL , Chan CH , Sands MS , Pearce DA , Cooper JD , Gillingwater TH |
| Ref : Hum Mol Genet , 18 :4066 , 2009 |
| Abstract : Kielar_2009_Hum.Mol.Genet_18_4066 |
| ESTHER : Kielar_2009_Hum.Mol.Genet_18_4066 |
| PubMedSearch : Kielar_2009_Hum.Mol.Genet_18_4066 |
| PubMedID: 19640925 |
| Gene_locus related to this paper: mouse-ppt |