Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease von Xu, Liwen, Lahiri, Premanjali, Skowronski, Jason, Bhatia, Neehar, Lattanzi, Annalisa, Porteus, Matthew H.

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Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αβ T cell-depleted haploidentical hematopoietic stem cell trans... von Wiebking, Volker, Lee, Ciaran M, Mostrel, Nathalie, Lahiri, Premanjali, Bak, Rasmus, Bao, Gang, Roncarolo, Maria Grazia, Bertaina, Alice, Porteus, Matthew H

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Genome editing of donor-derived T cells to generate allogeneic chimeric antigen receptor-modified T cells: optimizing alpha beta T-cell-depleted haploidentical hematopoietic stem c... von Wiebking, Volker, Lee, Ciaran M., Mostrel, Nathalie, Lahiri, Premanjali, Bak, Rasmus, Bao, Gang, Roncarolo, Maria Grazia, Bertaina, Alice, Porteus, Matthew H.

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Cedar Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe S... von Kanter, Julie, DiPersio, John F., Leavey, Patrick, Shyr, David C., Thompson, Alexis A, Porteus, Matthew H., Intondi, Allison, Lahiri, Premanjali, Dever, Daniel P., Petrusich, Alexandria, Lehrer-Graiwer, Joshua

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One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease von Shyr, David C, Lowsky, Robert, Miller, Weston, Schroeder, Mark A., Buchholz, Tonia, Dougall, Kirstin, Intondi, Allison, Charles, Alexandra, Lehrer, Josh, Bouge, Ali, Wolf, Stacey, MacDonald, Blair, Din, Hena, Lerner, Alana, Amoury, Manal, Treusch, Sebastien, Chew, Glen, Silva, Brian, Mashhedi, Haider, Siu, Vincent, Perrone, Ian, Vijay, Twaritha, Jain, Aayami, Krassovsky, Kristina, Matern, William, Lahiri, Premanjali, Rodriguez, Ryan, Skowronski, Jason, Batish, Arpit, Margittai, Dana, Wani, Prachi, Van Horn, Timothy, Flautero, Claudia, Flores, Rosalva, Lee, Jade, Tate, Keri, Roncarolo, Maria-Grazia, Feldman, Steven, DiPersio, John, Porteus, Matthew

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Genome editing of donor-derived T cells to generate allogeneic chimeric antigen receptor-modified T cells: optimizing αβ T-cell-depleted haploidentical hematopoietic stem cell tran... von Wiebking, Volker, Lee, Ciaran M., Mostrel, Nathalie, Lahiri, Premanjali, Bak, Rasmus, Bao, Gang, Roncarolo, Maria Grazia, Bertaina, Alice, Porteus, Matthew H.

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Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair von Perez-Bermejo, Juan A., Efagene, Oghene, Matern, William M., Holden, Jeffrey K., Kabir, Shaheen, Chew, Glen M., Andreoletti, Gaia, Catton, Eniola, Ennis, Craig L., Garcia, Angelica, Gerstenberg, Trevor L., Hill, Kaisle A., Jain, Aayami, Krassovsky, Kristina, Lalisan, Cassandra D., Lord, Daniel, Quejarro, B. Joy, Sales-Lee, Jade, Shah, Meet, Silva, Brian J., Skowronski, Jason, Strukov, Yuri G., Thomas, Joshua, Veraz, Michael, Vijay, Twaritha, Wallace, Kirby A., Yuan, Yue, Grogan, Jane L., Wienert, Beeke, Lahiri, Premanjali, Treusch, Sebastian, Dever, Daniel P., Soros, Vanessa B., Partridge, James R., Seim, Kristen L.

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Αβ T-Cell/CD19 B-Cell Depleted Haploidentical Stem Cell Transplantation: A New Platform for Curing Rare and Monogenic Disorders von Bertaina, Alice, Bacchetta, Rosa, Lewis, David B., Grimm, Paul C., Shah, Ami J., Agarwal, Rajni, Concepcion, Waldo, Czechowicz, Agnieszka, Bhatia, Neehar, Lahiri, Premanjali, Weinberg, Kenneth I, Parkman, Robertson, Porteus, Matthew, Roncarolo, Maria Grazia

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METHODS FOR IMPROVED PRODUCTION OF PRIMARY CD34+ CELLS von LAHIRI, Premanjali, LATTANZI, Annalisa, BHATIA, Neehar, PORTEUS, Matthew H, DEVER, Daniel P

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