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Molecular Therapy : the Journal of the American Society of Gene Therapy|March 8, 2021
Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase ComplexGustavo de Alencastro, Francesco Puzzo, Mara Pavel-Dinu, et al.Leukemia|March 16, 2023
Impact of U2AF1 mutations on circular RNA expression in myelodysplastic neoplasmsEileen Wedge, Ulvi Ahmadov, Thomas B Hansen, et al.Nature Communications|December 15, 2019
Functional significance of U2AF1 S34F mutations in lung adenocarcinomasMohammad S Esfahani, Luke J Lee, Young-Jun Jeon, et al.Nature Communications|March 31, 2025
Multilayered HIV-1 resistance in HSPCs through CCR5 Knockout and B cell secretion of HIV-inhibiting antibodiesWilliam N Feist, Sofia E Luna, Kaya Ben-Efraim, et al.Molecular Therapy. Advances|July 13, 2026
AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and qualitySujan Ravendran, Simon Fammé, Maya Graham Noer, et al.Nature Communications|January 29, 2025
Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesisAadit P Shah, Kiran R Majeti, Freja K Ekman, et al.Gene Therapy|March 13, 2021
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repairColin L Sweeney, Mara Pavel-Dinu, Uimook Choi, et al.Cell Reports|March 12, 2025
Distinctive CD8+ T cell activation by antigen-presenting plasmacytoid dendritic cells compared to conventional dendritic cellsRenée M van der Sluis, Juan L García-Rodríguez, Ian Helstrup Nielsen, et al.Blood|October 8, 2024
Failure of metabolic checkpoint control during late-stage granulopoiesis drives neutropenia in reticular dysgenesisWenqing Wang, Martin Arreola, Thomas Mathews, et al.Nature Biotechnology|August 3, 2023
High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibitionSridhar Selvaraj, William N Feist, Sebastien Viel, et al.Pageof 20