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CRISPR activation for SCN2A-related neurodevelopmental disorders

Serena Tamura, Andrew D. Nelson, Perry W.E. Spratt, Elizabeth C. Hamada, Xujia Zhou, Henry Kyoung, Zizheng Li, Coline Arnould, Vladyslav Barskyi, Beniamin Krupkin, Kiana Young, Jingjing Zhao, Stephanie S. Holden, Atehsa Sahagun, Caroline M. Keeshen, Congyi Lu, Roy Ben‐Shalom, Sunrae E. Taloma, Selin Schamiloglu, Ying C. Li, Lia Min, Paul M. Jenkins, Jen Q. Pan, Jeanne T. Paz, Stephan Sanders, Navneet Matharu, Nadav Ahituv, Kevin J. Bender

2025Nature17 citationsDOI

Topics & Concepts

HaploinsufficiencyGeneCRISPRPhenotypeBiologyGeneticsMedicineNeurodevelopmental disorderAutismDownregulation and upregulationMutationAutism spectrum disorderNeuroscienceBioinformaticsRegulation of gene expressionCRISPR and Genetic EngineeringGenetics and Neurodevelopmental DisordersAutism Spectrum Disorder Research
CRISPR activation for SCN2A-related neurodevelopmental disorders | Litcius