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Articles, Neurobiology of Disease

Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome

Saurabh K. Garg, Daniel T. Lioy, Hélène Cheval, James C. McGann, John M. Bissonnette, Matthew J. Murtha, Kevin D. Foust, Brian K. Kaspar, Adrian Bird and Gail Mandel
Journal of Neuroscience 21 August 2013, 33 (34) 13612-13620; DOI: https://doi.org/10.1523/JNEUROSCI.1854-13.2013
Saurabh K. Garg
1Vollum Institute and
2Howard Hughes Medical Institute, Oregon Health and Science University, Portland, Oregon 97239,
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Daniel T. Lioy
1Vollum Institute and
2Howard Hughes Medical Institute, Oregon Health and Science University, Portland, Oregon 97239,
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Hélène Cheval
3Wellcome Trust Centre for Cell Biology, University of Edinburgh, Mayfield Road, Edinburgh EH9 3JR, United Kingdom,
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James C. McGann
1Vollum Institute and
2Howard Hughes Medical Institute, Oregon Health and Science University, Portland, Oregon 97239,
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John M. Bissonnette
4Department of Cell and Developmental Biology and
5Department of Obstetrics and Gynecology, Oregon Health and Science University, Portland, Oregon 97239,
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Matthew J. Murtha
7Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Columbus, Ohio 43205
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Kevin D. Foust
6Department of Neuroscience, Ohio State University, Columbus, Ohio 43205, and
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Brian K. Kaspar
7Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Columbus, Ohio 43205
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Adrian Bird
3Wellcome Trust Centre for Cell Biology, University of Edinburgh, Mayfield Road, Edinburgh EH9 3JR, United Kingdom,
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Gail Mandel
1Vollum Institute and
2Howard Hughes Medical Institute, Oregon Health and Science University, Portland, Oregon 97239,
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Abstract

De novo mutations in the X-linked gene encoding the transcription factor methyl-CpG binding protein 2 (MECP2) are the most frequent cause of the neurological disorder Rett syndrome (RTT). Hemizygous males usually die of neonatal encephalopathy. Heterozygous females survive into adulthood but exhibit severe symptoms including microcephaly, loss of purposeful hand motions and speech, and motor abnormalities, which appear after a period of apparently normal development. Most studies have focused on male mouse models because of the shorter latency to and severity in symptoms, yet how well these mice mimic the disease in affected females is not clear. Very few therapeutic treatments have been proposed for females, the more gender-appropriate model. Here, we show that self-complementary AAV9, bearing MeCP2 cDNA under control of a fragment of its own promoter (scAAV9/MeCP2), is capable of significantly stabilizing or reversing symptoms when administered systemically into female RTT mice. To our knowledge, this is the first potential gene therapy for females afflicted with RTT.

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The Journal of Neuroscience: 33 (34)
Journal of Neuroscience
Vol. 33, Issue 34
21 Aug 2013
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Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome
Saurabh K. Garg, Daniel T. Lioy, Hélène Cheval, James C. McGann, John M. Bissonnette, Matthew J. Murtha, Kevin D. Foust, Brian K. Kaspar, Adrian Bird, Gail Mandel
Journal of Neuroscience 21 August 2013, 33 (34) 13612-13620; DOI: 10.1523/JNEUROSCI.1854-13.2013

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Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome
Saurabh K. Garg, Daniel T. Lioy, Hélène Cheval, James C. McGann, John M. Bissonnette, Matthew J. Murtha, Kevin D. Foust, Brian K. Kaspar, Adrian Bird, Gail Mandel
Journal of Neuroscience 21 August 2013, 33 (34) 13612-13620; DOI: 10.1523/JNEUROSCI.1854-13.2013
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