MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy

Mojgan Rastegar; Akitsu Hotta; Peter Pasceri; Maisam Makarem; Aaron Y. L. Cheung; Shauna Elliott; Katya J. Park; Megumi Adachi; Frederick S. Jones; Ian D. Clarke; Peter Dirks; James Ellis

doi:10.1371/journal.pone.0006810

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Open Access

Peer-reviewed

Research Article

MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy

Mojgan Rastegar,

Current address: Regenerative Medicine Program and Department of Biochemistry and Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada

Affiliation Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
⨯
Akitsu Hotta,

Affiliation Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
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Peter Pasceri,

Affiliation Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
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Maisam Makarem,

Affiliation Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
⨯
Aaron Y. L. Cheung,

Affiliations Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada, Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada
⨯
Shauna Elliott,

Affiliation Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
⨯
Katya J. Park,

Affiliations Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada, Institute of Medical Science, University of Toronto, Toronto, Ontario, Canada
⨯
Megumi Adachi,

Affiliation Experimental Neurobiology, The Neurosciences Institute, San Diego, California, United States of America
⨯
Frederick S. Jones,

Affiliation Experimental Neurobiology, The Neurosciences Institute, San Diego, California, United States of America
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Ian D. Clarke,

Affiliation Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
⨯
Peter Dirks,

Affiliation Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
⨯
James Ellis

* E-mail: jellis@sickkids.ca

Affiliations Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada, Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada
⨯

MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy

Mojgan Rastegar,
Akitsu Hotta,
Peter Pasceri,
Maisam Makarem,
Aaron Y. L. Cheung,
Shauna Elliott,
Katya J. Park,
Megumi Adachi,
Frederick S. Jones,
Ian D. Clarke

Published: August 27, 2009
https://doi.org/10.1371/journal.pone.0006810

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