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CRISPR activation rescues abnormalities in SCN2A haploinsufficiency-associated autism spectrum disorder
Serena Tamura, Andrew D. Nelson, Perry W.E. Spratt, Henry Kyoung, Xujia Zhou, Zizheng Li, Jingjing Zhao, Stephanie S. Holden, Atehsa Sahagun, Caroline M. Keeshen, Congyi Lu, Elizabeth C. Hamada, Roy Ben-Shalom, Jen Q. Pan, Jeanne T. Paz, View ORCID ProfileStephan J. Sanders, View ORCID ProfileNavneet Matharu, View ORCID ProfileNadav Ahituv, View ORCID ProfileKevin J. Bender
doi: https://doi.org/10.1101/2022.03.30.486483
Serena Tamura
1Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA
2Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA
Andrew D. Nelson
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
Perry W.E. Spratt
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
Henry Kyoung
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
Xujia Zhou
1Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA
2Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA
Zizheng Li
1Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA
2Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA
Jingjing Zhao
1Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA
2Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA
Stephanie S. Holden
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
6Gladstone Institute of Neurological Disease, Gladstone Institutes, San Francisco CA, USA
Atehsa Sahagun
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
Caroline M. Keeshen
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
Congyi Lu
7Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA
Elizabeth C. Hamada
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
Roy Ben-Shalom
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
Jen Q. Pan
7Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA
Jeanne T. Paz
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
6Gladstone Institute of Neurological Disease, Gladstone Institutes, San Francisco CA, USA
Stephan J. Sanders
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
5Department of Psychiatry, University of California San Francisco, San Francisco, CA, USA
Navneet Matharu
1Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA
2Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA
Nadav Ahituv
1Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA
2Institute for Human Genetics, University of California San Francisco, San Francisco, CA, USA
Kevin J. Bender
3Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA
4Department of Neurology, University of California San Francisco, San Francisco, CA, USA
Posted April 01, 2022.
CRISPR activation rescues abnormalities in SCN2A haploinsufficiency-associated autism spectrum disorder
Serena Tamura, Andrew D. Nelson, Perry W.E. Spratt, Henry Kyoung, Xujia Zhou, Zizheng Li, Jingjing Zhao, Stephanie S. Holden, Atehsa Sahagun, Caroline M. Keeshen, Congyi Lu, Elizabeth C. Hamada, Roy Ben-Shalom, Jen Q. Pan, Jeanne T. Paz, Stephan J. Sanders, Navneet Matharu, Nadav Ahituv, Kevin J. Bender
bioRxiv 2022.03.30.486483; doi: https://doi.org/10.1101/2022.03.30.486483
CRISPR activation rescues abnormalities in SCN2A haploinsufficiency-associated autism spectrum disorder
Serena Tamura, Andrew D. Nelson, Perry W.E. Spratt, Henry Kyoung, Xujia Zhou, Zizheng Li, Jingjing Zhao, Stephanie S. Holden, Atehsa Sahagun, Caroline M. Keeshen, Congyi Lu, Elizabeth C. Hamada, Roy Ben-Shalom, Jen Q. Pan, Jeanne T. Paz, Stephan J. Sanders, Navneet Matharu, Nadav Ahituv, Kevin J. Bender
bioRxiv 2022.03.30.486483; doi: https://doi.org/10.1101/2022.03.30.486483
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