748. Impact of Age of Treatment on AAVrh.10hCLN2-Mediated Amelioration of Symptoms in CLN2 Knockout Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1016/s1525-0016(16)44954-3 ◽

2007 ◽

Vol 15 ◽

pp. S287

Keyword(s):

Mouse Model ◽

Knockout Mouse ◽

Neuronal Ceroid Lipofuscinosis ◽

Knockout Mouse Model ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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Profound loss of GABAergic interneurons in the PPT1 knockout mouse model of infantile neuronal ceroid lipofuscinosis

Neuropathology and Applied Neurobiology ◽

10.1046/j.1365-2990.2002.39286_29.x ◽

2002 ◽

Vol 28 (2) ◽

pp. 158-159 ◽

Author(s):

J. D. Cooper ◽

P. Gupta ◽

E. Bible ◽

S. Hofmann ◽

P. Lantos) ◽

...

Keyword(s):

Mouse Model ◽

Knockout Mouse ◽

Neuronal Ceroid Lipofuscinosis ◽

Gabaergic Interneurons ◽

Knockout Mouse Model ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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Retinal Pathology and Function in a Cln3 Knockout Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis (Batten Disease)

Molecular and Cellular Neuroscience ◽

10.1006/mcne.2001.1099 ◽

2002 ◽

Vol 19 (4) ◽

pp. 515-527 ◽

Author(s):

Gail M. Seigel ◽

Andrew Lotery ◽

Ari Kummer ◽

David J. Bernard ◽

Nicholas D.E. Greene ◽

...

Keyword(s):

Mouse Model ◽

Knockout Mouse ◽

Neuronal Ceroid Lipofuscinosis ◽

Batten Disease ◽

Juvenile Neuronal Ceroid Lipofuscinosis ◽

Knockout Mouse Model ◽

Ceroid Lipofuscinosis ◽

Retinal Pathology ◽

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Morphologic and Functional Correlates of Synaptic Pathology in the Cathepsin D Knockout Mouse Model of Congenital Neuronal Ceroid Lipofuscinosis

Journal of Neuropathology & Experimental Neurology ◽

10.1097/nen.0b013e318238fc28 ◽

2011 ◽

Vol 70 (12) ◽

pp. 1089-1096 ◽

Author(s):

Sabine Koch ◽

Svetlana M. Molchanova ◽

Ann K. Wright ◽

Andrew Edwards ◽

Jonathan D. Cooper ◽

...

Keyword(s):

Mouse Model ◽

Cathepsin D ◽

Knockout Mouse ◽

Neuronal Ceroid Lipofuscinosis ◽

Knockout Mouse Model ◽

Ceroid Lipofuscinosis ◽

Synaptic Pathology

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218. Gene Therapy and Enzyme Replacement in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1016/j.ymthe.2006.08.243 ◽

2006 ◽

Vol 13 ◽

pp. S84

Author(s):

Michael Chang ◽

David Sleat ◽

Seng Cheng ◽

Marco Passini ◽

Peter Lobel ◽

...

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Neuronal Ceroid Lipofuscinosis ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Enzyme Replacement ◽

Ceroid Lipofuscinosis

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The blood-brain barrier is disrupted in a mouse model of infantile neuronal ceroid lipofuscinosis: amelioration by resveratrol

Human Molecular Genetics ◽

10.1093/hmg/dds038 ◽

2012 ◽

Vol 21 (10) ◽

pp. 2233-2244 ◽

Author(s):

Arjun Saha ◽

Chinmoy Sarkar ◽

Satya P. Singh ◽

Zhongjian Zhang ◽

Jeeva Munasinghe ◽

...

Keyword(s):

Mouse Model ◽

Blood Brain Barrier ◽

Neuronal Ceroid Lipofuscinosis ◽

Brain Barrier ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis ◽

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Intrathecal and intravenous combination gene therapy in the mouse model of infantile neuronal ceroid lipofuscinosis extends lifespan and improves behavioral outcomes in moderately affected mice

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2018.12.149 ◽

2019 ◽

Vol 126 (2) ◽

pp. S63-S64

Author(s):

Steven J. Gray ◽

Alejandra Rozenberg ◽

Erik Lykken ◽

Linas Padegimas ◽

Timothy Miller

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Neuronal Ceroid Lipofuscinosis ◽

Behavioral Outcomes ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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Gene expression profiling in a mouse model of infantile neuronal ceroid lipofuscinosis reveals upregulation of immediate early genes and mediators of the inflammatory response

BMC Neuroscience ◽

10.1186/1471-2202-8-95 ◽

2007 ◽

Vol 8 (1) ◽

pp. 95 ◽

Author(s):

Xingwen Qiao ◽

Jui-Yun Lu ◽

Sandra L Hofmann

Keyword(s):

Gene Expression ◽

Inflammatory Response ◽

Mouse Model ◽

Gene Expression Profiling ◽

Expression Profiling ◽

Neuronal Ceroid Lipofuscinosis ◽

Immediate Early Genes ◽

Immediate Early ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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427. AAV2- and AAV5-Mediated CNS Delivery of Human CLN2 Reduces Lysosomal Storage in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1016/j.ymthe.2005.06.429 ◽

2005 ◽

Vol 11 ◽

pp. S166

Keyword(s):

Mouse Model ◽

Neuronal Ceroid Lipofuscinosis ◽

Lysosomal Storage ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis ◽

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Regional and cellular neuropathology in the palmitoyl protein thioesterase-1 null mutant mouse model of infantile neuronal ceroid lipofuscinosis

Neurobiology of Disease ◽

10.1016/j.nbd.2004.02.010 ◽

2004 ◽

Vol 16 (2) ◽

pp. 346-359 ◽

Author(s):

Ellen Bible ◽

Praveena Gupta ◽

Sandra L Hofmann ◽

Jonathan D Cooper

Keyword(s):

Mouse Model ◽

Mutant Mouse ◽

Neuronal Ceroid Lipofuscinosis ◽

Null Mutant ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis ◽

Palmitoyl Protein Thioesterase ◽

Null Mutant Mouse

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Intraventricular Enzyme Replacement Improves Disease Phenotypes in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1038/mt.2008.9 ◽

2008 ◽

Vol 16 (4) ◽

pp. 649-656 ◽

Author(s):

Michael Chang ◽

Jonathan D Cooper ◽

David E Sleat ◽

Seng H Cheng ◽

James C Dodge ◽

...

Keyword(s):

Mouse Model ◽

Neuronal Ceroid Lipofuscinosis ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Enzyme Replacement ◽

Disease Phenotypes ◽

Ceroid Lipofuscinosis

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