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Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of Spinal Muscular Atrophy
by
Glascock, Jacqueline J.
,
Shababi, Monir
,
Wetz, Mary J.
,
Krogman, Megan M.
,
Lorson, Christian L.
Published in
Biochemical and biophysical research communications
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Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy
by
Cobb, Melissa S
,
Rose, Ferril F
,
Rindt, Hansjörg
,
Glascock, Jacqueline J
,
Shababi, Monir
,
Miller, Madeline R
,
Osman, Erkan Y
,
Yen, Pei-Fen
,
Garcia, Michael L
,
Martin, Brittanie R
,
Wetz, Mary J
,
Mazzasette, Chiara
,
Feng, Zhihua
,
Ko, Chien-Ping
,
Lorson, Christian L
Published in
Human molecular genetics
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Biochemical And Biophysical Research Communications
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Human Molecular Genetics
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Animals
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Biochemistry & Molecular Biology
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Disease Models, Animal
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Life Sciences & Biomedicine
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Mice
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Muscular Atrophy, Spinal - Pathology
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Science & Technology
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Survival Of Motor Neuron 1 Protein - Genetics
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Survival Of Motor Neuron 2 Protein - Genetics
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Animal Models
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Biophysics
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Body Weight
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Brain - Metabolism
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Cell Survival
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Central Nervous System
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Dependovirus
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Exons
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Gene Expression Regulation
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Gene Therapy
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Gene Transfer Techniques
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