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

Steven J. Gray; Alejandra Rozenberg; Erik Lykken; Linas Padegimas; Timothy Miller

doi:10.1016/j.ymgme.2018.12.149

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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Combination intrathecal and intravenous gene therapy reveals a dominant role for treatment age in determining survival and behavioral outcomes in the mouse model of infantile neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.11.265 ◽

2020 ◽

Vol 129 (2) ◽

pp. S104

Author(s):

Erik A. Lykken ◽

Alejandra J. Rozenberg ◽

Steven J. Gray

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Neuronal Ceroid Lipofuscinosis ◽

Dominant Role ◽

Behavioral Outcomes ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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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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Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2017.12.338 ◽

2018 ◽

Vol 123 (2) ◽

pp. S124 ◽

Cited By ~ 2

Author(s):

Alejandra J. Rozenberg ◽

Erik Lykken ◽

Kaye Spratt ◽

Timothy J. Miller ◽

Steven J. Gray

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Neuronal Ceroid Lipofuscinosis ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Ceroid Lipofuscinosis

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Early intrathecal gene therapy extends lifespan and improves quality of life in a mouse model for infantile neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2016.11.301 ◽

2017 ◽

Vol 120 (1-2) ◽

pp. S117 ◽

Cited By ~ 1

Author(s):

Alejandra J Rozenberg ◽

Steven J Gray

Keyword(s):

Quality Of Life ◽

Gene Therapy ◽

Mouse Model ◽

Neuronal Ceroid Lipofuscinosis ◽

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 ◽

Cited By ~ 7

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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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 ◽

Cited By ~ 41

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 ◽

Blood Brain

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224. Comparison of AAV Serotypes 2 and 5 Based Vectors for Corrective Gene Therapy for Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1016/s1525-0016(16)40666-0 ◽

2003 ◽

Vol 7 (5) ◽

pp. S89

Keyword(s):

Gene Therapy ◽

Neuronal Ceroid Lipofuscinosis ◽

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 ◽

Cited By ~ 21

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 ◽

Cns Delivery

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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 ◽

Cited By ~ 121

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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