Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies
Sofia B. Lizarraga, Li Ma, Abbie M. Maguire, Laura I. van Dyck, Qing Wu, Qing Ouyang, Brian C. Kavanaugh, Dipal V. Nagda, Liane L. Livi, Matthew F. Pescosolido, Michael Schmidt, Shanique B. Alabi, Mara H. Cowen, Paul Brito‐Vargas, Diane Hoffman–Kim, , Eric M. Morrow
The paper uses iPSC-derived neurons from Christianson syndrome patients to show allele-specific responses to rescue strategies, highlighting the need for personalized therapeutic approaches.
Science Translational Medicine · Feb 10, 2021Peer-reviewedNeuroscience