| Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease A Lattanzi, J Camarena, P Lahiri, H Segal, W Srifa, CA Vakulskas, ... Science translational medicine 13 (598), eabf2444, 2021 | 105 | 2021 |
| 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 … V Wiebking, CM Lee, N Mostrel, P Lahiri, R Bak, G Bao, MG Roncarolo, ... Haematologica 106 (3), 847, 2021 | 54 | 2021 |
| 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 … J Kanter, JF DiPersio, P Leavey, DC Shyr, AA Thompson, MH Porteus, ... Blood 138, 1864, 2021 | 9 | 2021 |
| Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease L Xu, P Lahiri, J Skowronski, N Bhatia, A Lattanzi, MH Porteus Molecular Therapy-Methods & Clinical Development 30, 317-331, 2023 | 3 | 2023 |
| αβ T-Cell/CD19 B-Cell Depleted Haploidentical Stem Cell Transplantation: A New Platform for Curing Rare and Monogenic Disorders A Bertaina, R Bacchetta, DB Lewis, PC Grimm, AJ Shah, R Agarwal, ... Biology of Blood and Marrow Transplantation 26 (3), S288, 2020 | 2 | 2020 |
| Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair JA Perez-Bermejo, O Efagene, WM Matern, JK Holden, S Kabir, GM Chew, ... Nature Communications 15 (1), 2625, 2024 | | 2024 |
| 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 DC Shyr, R Lowsky, W Miller, MA Schroeder, T Buchholz, K Dougall, ... Blood 142, 5000, 2023 | | 2023 |
| 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 … JF Di Persio, J Kanter, P Leavey, DC Shyr, AA Thompson, MH Porteus, ... MOLECULAR THERAPY 30 (4), 379-379, 2022 | | 2022 |
| Towards The Clinical Translation of Gene Correction in Hematopoietic Stem Cells for Sickle Cell Disease Treatment A Lattanzi, DP Dever, J Camarena, P Lahiri, H Segal, N Talbott, W Srifa, ... MOLECULAR THERAPY 27 (4), 448-448, 2019 | | 2019 |
