Gene therapy for congenital marrow failure syndromes – no longer grasping at straws?

por Voit, Richard A., Corey, Seth J.
Publicado 2023

Pyruvate kinase deficiency in a newborn with extramedullary hematopoiesis in the skin

por Voit, Richard A., Grace, Rachael F.
Publicado 2020

Nuclease-mediated gene editing by homologous recombination of the human globin locus

por Voit, Richard A., Hendel, Ayal, Pruett-Miller, Shondra M., Porteus, Matthew H.
Publicado 2014

Pathogenic BCL11A variants provide insights into the mechanisms of human fetal hemoglobin silencing

por Shen, Yong, Li, Rick, Teichert, Kristian, Montbleau, Kara E., Verboon, Jeffrey M., Voit, Richard A., Sankaran, Vijay G.
Publicado 2021

CD11c regulates neutrophil maturation

por Hou, Lifei, Voit, Richard A., Shibamura-Fujiogi, Miho, Koutsogiannaki, Sophia, Li, Yunan, Chen, Yue, Luo, Hongbo, Sankaran, Vijay G., Yuki, Koichi
Publicado 2022

SAPTA: a new design tool for improving TALE nuclease activity

por Lin, Yanni, Fine, Eli J., Zheng, Zhilan, Antico, Christopher J., Voit, Richard A., Porteus, Matthew H., Cradick, Thomas J., Bao, Gang
Publicado 2014

A genetic disorder reveals a hematopoietic stem cell regulatory network co-opted in leukemia

por Voit, Richard A., Tao, Liming, Yu, Fulong, Cato, Liam D., Cohen, Blake, Fleming, Travis J., Antoszewski, Mateusz, Liao, Xiaotian, Fiorini, Claudia, Nandakumar, Satish K., Wahlster, Lara, Teichert, Kristian, Regev, Aviv, Sankaran, Vijay G.
Publicado 2022

A unified model of human hemoglobin switching through single-cell genome editing

por Shen, Yong, Verboon, Jeffrey M., Zhang, Yuannyu, Liu, Nan, Kim, Yoon Jung, Marglous, Samantha, Nandakumar, Satish K., Voit, Richard A., Fiorini, Claudia, Ejaz, Ayesha, Basak, Anindita, Orkin, Stuart H., Xu, Jian, Sankaran, Vijay G.
Publicado 2021

Familial thrombocytopenia due to a complex structural variant resulting in a WAC-ANKRD26 fusion transcript

por Wahlster, Lara, Verboon, Jeffrey M., Ludwig, Leif S., Black, Susan C., Luo, Wendy, Garg, Kopal, Voit, Richard A., Collins, Ryan L., Garimella, Kiran, Costello, Maura, Chao, Katherine R., Goodrich, Julia K., DiTroia, Stephanie P., O’Donnell-Luria, Anne, Talkowski, Michael E., Michelson, Alan D., Cantor, Alan B., Sankaran, Vijay G.
Publicado 2021