Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair

por Sweeney, Colin L., Pavel-Dinu, Mara, Choi, Uimook, Brault, Julie, Liu, Taylor, Koontz, Sherry, Li, Linhong, Theobald, Narda, Lee, Janet, Bello, Ezekiel A., Wu, Xiaolin, Meis, Ronald J., Dahl, Gary A., Porteus, Matthew H., Malech, Harry L., See De Ravin, Suk
Publicado 2021

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells

por Brault, Julie, Liu, Taylor, Liu, Siyuan, Lawson, Amanda, Choi, Uimook, Kozhushko, Nikita, Bzhilyanskaya, Vera, Pavel-Dinu, Mara, Meis, Ronald J., Eckhaus, Michael A., Burkett, Sandra S., Bosticardo, Marita, Kleinstiver, Benjamin P., Notarangelo, Luigi D., Lazzarotto, Cicera R., Tsai, Shengdar Q., Wu, Xiaolin, Dahl, Gary A., Porteus, Matthew H., Malech, Harry L., De Ravin, Suk See
Publicado 2023