The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells

por Romero, Zulema, Campo-Fernandez, Beatriz, Wherley, Jennifer, Kaufman, Michael L, Urbinati, Fabrizia, Cooper, Aaron R, Hoban, Megan D, Baldwin, Kismet M, Lumaquin, Dianne, Wang, Xiaoyan, Senadheera, Shantha, Hollis, Roger P, Kohn, Donald B
Publicado 2015

Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells

por Benitez, Elizabeth K., Lomova Kaufman, Anastasia, Cervantes, Lilibeth, Clark, Danielle N., Ayoub, Paul G., Senadheera, Shantha, Osborne, Kyle, Sanchez, Julie M., Crisostomo, Ralph Valentine, Wang, Xiaoyan, Reuven, Nina, Shaul, Yosef, Hollis, Roger P., Romero, Zulema, Kohn, Donald B.
Publicado 2020

High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation

por Magis, Wendy, DeWitt, Mark A., Wyman, Stacia K., Vu, Jonathan T., Heo, Seok-Jin, Shao, Shirley J., Hennig, Finn, Romero, Zulema G., Campo-Fernandez, Beatriz, Said, Suzanne, McNeill, Matthew S., Rettig, Garrett R., Sun, Yongming, Wang, Yu, Behlke, Mark A., Kohn, Donald B., Boffelli, Dario, Walters, Mark C., Corn, Jacob E., Martin, David I.K.
Publicado 2022

Supramolecular nanosubstrate–mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies

por Yang, Peng, Chou, Shih-Jie, Li, Jindian, Hui, Wenqiao, Liu, Wenfei, Sun, Na, Zhang, Ryan Y., Zhu, Yazhen, Tsai, Ming-Long, Lai, Henkie I., Smalley, Matthew, Zhang, Xinyue, Chen, Jiayuan, Romero, Zulema, Liu, Dahai, Ke, Zunfu, Zou, Chang, Lee, Chin-Fa, Jonas, Steven J., Ban, Qian, Weiss, Paul S., Kohn, Donald B., Chen, Kai, Chiou, Shih-Hwa, Tseng, Hsian-Rong
Publicado 2020