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Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies

View ORCID ProfileSofia B. Lizarraga, Abbie M. Maguire, Li Ma, Laura I. van Dyck, Qing Wu, Dipal Nagda, Liane L. Livi, Matthew F. Pescosolido, Michael Schmidt, Shanique Alabi, Mara H. Cowen, Paul Brito-Vargas, Diane Hoffman-Kim, Ece D. Gamsiz Uzun, Avner Schlessinger, Richard N. Jones, View ORCID ProfileEric M. Morrow
doi: https://doi.org/10.1101/444232
Sofia B. Lizarraga
1Department of Biological Sciences, University of South Carolina, Columbia, South Carolina, USA
2Center for Childhood Neurotherapeutics, University of South Carolina, Columbia, South Carolina, USA
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  • ORCID record for Sofia B. Lizarraga
Abbie M. Maguire
3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
4Hassenfeld Child Health Innovation Institute, Brown University, Providence, Rhode Island, USA
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Li Ma
3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
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Laura I. van Dyck
3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
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Qing Wu
3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
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Dipal Nagda
3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
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Liane L. Livi
5Department of Molecular Pharmacology, Physiology, and Biotechnology, Brown University, Providence, Rhode Island, USA
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Matthew F. Pescosolido
3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
4Hassenfeld Child Health Innovation Institute, Brown University, Providence, Rhode Island, USA
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Michael Schmidt
3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
4Hassenfeld Child Health Innovation Institute, Brown University, Providence, Rhode Island, USA
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Shanique Alabi
6Department of Pharmacological Sciences, Icahn School of Medicine at Mount Sinai, New York, New York, USA
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Mara H. Cowen
1Department of Biological Sciences, University of South Carolina, Columbia, South Carolina, USA
2Center for Childhood Neurotherapeutics, University of South Carolina, Columbia, South Carolina, USA
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Paul Brito-Vargas
1Department of Biological Sciences, University of South Carolina, Columbia, South Carolina, USA
2Center for Childhood Neurotherapeutics, University of South Carolina, Columbia, South Carolina, USA
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Diane Hoffman-Kim
5Department of Molecular Pharmacology, Physiology, and Biotechnology, Brown University, Providence, Rhode Island, USA
7Carney Institute for Brain Science, Brown University, Providence, Rhode Island, USA
8Center for Biomedical Engineering, Brown University, Providence, Rhode Island, USA
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Ece D. Gamsiz Uzun
9Department of Pathology and Laboratory Medicine, Warren Alpert Medical School of Brown University, Providence, Rhode Island, USA
10Center for Computational Molecular Biology, Brown University, Providence, Rhode Island, USA
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Avner Schlessinger
6Department of Pharmacological Sciences, Icahn School of Medicine at Mount Sinai, New York, New York, USA
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Richard N. Jones
11Department of Psychiatry and Human Behavior, Warren Alpert Medical School of Brown University, Providence, Rhode Island, USA
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Eric M. Morrow
3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
4Hassenfeld Child Health Innovation Institute, Brown University, Providence, Rhode Island, USA
7Carney Institute for Brain Science, Brown University, Providence, Rhode Island, USA
10Center for Computational Molecular Biology, Brown University, Providence, Rhode Island, USA
11Department of Psychiatry and Human Behavior, Warren Alpert Medical School of Brown University, Providence, Rhode Island, USA
12Developmental Disorders Genetics Research Program, Emma Pendleton Bradley Hospital, East Providence, Rhode Island, USA
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  • ORCID record for Eric M. Morrow
  • For correspondence: eric_morrow@brown.edu
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Article Information

doi 
https://doi.org/10.1101/444232
History 
  • October 16, 2018.
Copyright 
The copyright holder for this preprint is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. All rights reserved. No reuse allowed without permission.

Author Information

  1. Sofia B. Lizarraga1,2,13,
  2. Abbie M. Maguire3,4,13,
  3. Li Ma3,
  4. Laura I. van Dyck3,
  5. Qing Wu3,
  6. Dipal Nagda3,
  7. Liane L. Livi5,
  8. Matthew F. Pescosolido3,4,
  9. Michael Schmidt3,4,
  10. Shanique Alabi6,
  11. Mara H. Cowen1,2,
  12. Paul Brito-Vargas1,2,
  13. Diane Hoffman-Kim5,7,8,
  14. Ece D. Gamsiz Uzun9,10,
  15. Avner Schlessinger6,
  16. Richard N. Jones11 and
  17. Eric M. Morrow3,4,7,10,11,12,13,*
  1. 1Department of Biological Sciences, University of South Carolina, Columbia, South Carolina, USA
  2. 2Center for Childhood Neurotherapeutics, University of South Carolina, Columbia, South Carolina, USA
  3. 3Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, Rhode Island, USA
  4. 4Hassenfeld Child Health Innovation Institute, Brown University, Providence, Rhode Island, USA
  5. 5Department of Molecular Pharmacology, Physiology, and Biotechnology, Brown University, Providence, Rhode Island, USA
  6. 6Department of Pharmacological Sciences, Icahn School of Medicine at Mount Sinai, New York, New York, USA
  7. 7Carney Institute for Brain Science, Brown University, Providence, Rhode Island, USA
  8. 8Center for Biomedical Engineering, Brown University, Providence, Rhode Island, USA
  9. 9Department of Pathology and Laboratory Medicine, Warren Alpert Medical School of Brown University, Providence, Rhode Island, USA
  10. 10Center for Computational Molecular Biology, Brown University, Providence, Rhode Island, USA
  11. 11Department of Psychiatry and Human Behavior, Warren Alpert Medical School of Brown University, Providence, Rhode Island, USA
  12. 12Developmental Disorders Genetics Research Program, Emma Pendleton Bradley Hospital, East Providence, Rhode Island, USA
  1. ↵*Correspondence and material requests should be addressed to: Eric M. Morrow, M.D. Ph.D., Laboratories for Molecular Medicine, Brown University, 70 Ship Street, Providence, Rhode Island 02912 USA, Tel: 401-863-9778, Fax: 401-432-1607 E-mail: eric_morrow{at}brown.edu
  1. ↵13 These authors contributed equally to this work.

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Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies
Sofia B. Lizarraga, Abbie M. Maguire, Li Ma, Laura I. van Dyck, Qing Wu, Dipal Nagda, Liane L. Livi, Matthew F. Pescosolido, Michael Schmidt, Shanique Alabi, Mara H. Cowen, Paul Brito-Vargas, Diane Hoffman-Kim, Ece D. Gamsiz Uzun, Avner Schlessinger, Richard N. Jones, Eric M. Morrow
bioRxiv 444232; doi: https://doi.org/10.1101/444232
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Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies
Sofia B. Lizarraga, Abbie M. Maguire, Li Ma, Laura I. van Dyck, Qing Wu, Dipal Nagda, Liane L. Livi, Matthew F. Pescosolido, Michael Schmidt, Shanique Alabi, Mara H. Cowen, Paul Brito-Vargas, Diane Hoffman-Kim, Ece D. Gamsiz Uzun, Avner Schlessinger, Richard N. Jones, Eric M. Morrow
bioRxiv 444232; doi: https://doi.org/10.1101/444232

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