| Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells SS De Ravin, J Brault, RJ Meis, S Liu, L Li, M Pavel-Dinu, CR Lazzarotto, ... Blood, The Journal of the American Society of Hematology 137 (19), 2598-2608, 2021 | 52 | 2021 |
| Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair CL Sweeney, M Pavel-Dinu, U Choi, J Brault, T Liu, S Koontz, L Li, ... Gene therapy 28 (6), 373-390, 2021 | 47 | 2021 |
| CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes J Brault, T Liu, E Bello, S Liu, CL Sweeney, RJ Meis, S Koontz, C Corsino, ... Blood, The Journal of the American Society of Hematology 138 (26), 2768-2780, 2021 | 26 | 2021 |
| Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency SS De Ravin, S Liu, CL Sweeney, J Brault, N Whiting-Theobald, M Ma, ... Nature communications 13 (1), 3710, 2022 | 17 | 2022 |
| MAGT1 messenger RNA-corrected autologous T and natural killer cells for potential cell therapy in X-linked immunodeficiency with magnesium defect, Epstein-Barr virus infection … J Brault, RJ Meis, L Li, E Bello, T Liu, CL Sweeney, SM Koontz, K Dowdell, ... Cytotherapy 23 (3), 203-210, 2021 | 10 | 2021 |
| CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells J Brault, T Liu, S Liu, A Lawson, U Choi, N Kozhushko, V Bzhilyanskaya, ... Frontiers in Immunology 13, 1067417, 2023 | 8 | 2023 |
| Enhanced transduction lentivector gene therapy for treatment of older patients with X-linked severe combined immunodeficiency SS De Ravin, SA O'Brien, N Kwatemaa, N Theobald, S Liu, J Lee, ... Blood 134, 608, 2019 | 8 | 2019 |
| NADPH oxidase correction by mRNA transfection of apheresis granulocytes in chronic granulomatous disease SS De Ravin, J Brault, RJ Meis, L Li, N Theobald, AC Bonifacino, H Lei, ... Blood Advances 4 (23), 5976-5987, 2020 | 4 | 2020 |
| Anna Villa, National Research Council (CNR), Italy F Staal, A Magnani, SS De Ravin, J Brault, T Liu, S Liu, A Lawson, U Choi, ... | | 2023 |
| CRISPR/Cas9 Targeted MAGT1 Gene Addition into XMEN Patient Hematopoietic Stem Cells and Peripheral Blood T Cells J Brault, T Liu, E Bello, RJ Meis, S Liu, S Koontz, C Corsino, U Choi, ... MOLECULAR THERAPY 28 (4), 408-409, 2020 | | 2020 |
| Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease J Brault, E Bello, RJ Meis, T Liu, SM Koontz, K Dowdell, L Li, C Allen, ... Blood 134, 4637, 2019 | | 2019 |
