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

Enzyme Replacement ◽

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

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 ◽

Behavioral Outcomes ◽

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 ◽

Cited By ~ 109

Author(s):

Michael Chang ◽

Jonathan D Cooper ◽

David E Sleat ◽

Seng H Cheng ◽

James C Dodge ◽

...

Keyword(s):

Mouse Model ◽

Enzyme Replacement ◽

Disease Phenotypes ◽

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

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 ◽

Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

10.1016/j.ymgme.2015.05.005 ◽

2015 ◽

Vol 116 (1-2) ◽

pp. 98-105 ◽

Cited By ~ 20

Author(s):

Jui-Yun Lu ◽

Hemanth R. Nelvagal ◽

Lingling Wang ◽

Shari G. Birnbaum ◽

Jonathan D. Cooper ◽

...

Keyword(s):

Mouse Model ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Motor Function ◽

Enzyme Replacement ◽

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

Molecular Therapy ◽

10.1038/sj.mt.6300415 ◽

2008 ◽

Author(s):

Michael Chang ◽

Jonathan D Cooper ◽

David E Sleat ◽

Seng H Cheng ◽

James C Dodge ◽

...

Keyword(s):

Mouse Model ◽

Enzyme Replacement ◽

Disease Phenotypes ◽

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

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 ◽

Dominant Role ◽

Behavioral Outcomes ◽

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

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 ◽

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

10.1016/j.ymgme.2012.05.009 ◽

2012 ◽

Vol 107 (1-2) ◽

pp. 213-221 ◽

Cited By ~ 26

Author(s):

Jie Hu ◽

Jui-Yun Lu ◽

Andrew M.S. Wong ◽

Linda S. Hynan ◽

Shari G. Birnbaum ◽

...

Keyword(s):

Mouse Model ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

High Dose ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

Ceroid Lipofuscinosis ◽

Palmitoyl Protein Thioesterase

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 ◽

Gabaergic Interneurons ◽

Knockout Mouse Model ◽

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 ◽

Brain Barrier ◽

Ceroid Lipofuscinosis ◽

Blood Brain