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Page 1
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.
Sweeney CL, Pavel-Dinu M, Choi U, Brault J, Liu T, Koontz S, Li L, Theobald N, Lee J, Bello EA, Wu X, Meis RJ, Dahl GA, Porteus MH, Malech HL, De Ravin SS. Sweeney CL, et al. Among authors: bello ea. Gene Ther. 2021 Jun;28(6):373-390. doi: 10.1038/s41434-021-00251-z. Epub 2021 Mar 12. Gene Ther. 2021. PMID: 33712802 Free PMC article.
CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.
Brault J, Liu T, Bello E, Liu S, Sweeney CL, Meis RJ, Koontz S, Corsino C, Choi U, Vayssiere G, Bosticardo M, Dowdell K, Lazzarotto CR, Clark AB, Notarangelo LD, Ravell JC, Lenardo MJ, Kleinstiver BP, Tsai SQ, Wu X, Dahl GA, Malech HL, De Ravin SS. Brault J, et al. Blood. 2021 Dec 30;138(26):2768-2780. doi: 10.1182/blood.2021011192. Blood. 2021. PMID: 34086870 Free PMC article.
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 and neoplasia disease.
Brault J, Meis RJ, Li L, Bello E, Liu T, Sweeney CL, Koontz SM, Dowdell K, Theobald N, Lee J, Allen C, Clark AB, Ravell JC, Lenardo MJ, Dahl GA, Malech HL, De Ravin SS. Brault J, et al. Cytotherapy. 2021 Mar;23(3):203-210. doi: 10.1016/j.jcyt.2020.08.013. Epub 2020 Oct 10. Cytotherapy. 2021. PMID: 33051095
Gene Editing Rescues In vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System.
Gardner CL, Pavel-Dinu M, Dobbs K, Bosticardo M, Reardon PK, Lack J, DeRavin SS, Le K, Bello E, Pala F, Delmonte OM, Malech H, Montel-Hagan A, Crooks G, Acuto O, Porteus MH, Notarangelo LD. Gardner CL, et al. J Clin Immunol. 2021 Jul;41(5):852-862. doi: 10.1007/s10875-021-00989-6. Epub 2021 Mar 1. J Clin Immunol. 2021. PMID: 33650026 Free PMC article.
Correction to: Gene Editing Rescues in Vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System.
Gardner CL, Pavel-Dinu M, Dobbs K, Bosticardo M, Reardon PK, Lack J, DeRavin SS, Le K, Bello E, Pala F, Delmonte OM, Malech H, Montel-Hagen A, Crooks G, Acuto O, Porteus MH, Notarangelo LD. Gardner CL, et al. J Clin Immunol. 2021 Jul;41(5):863. doi: 10.1007/s10875-021-01030-6. J Clin Immunol. 2021. PMID: 33821398 No abstract available.
Loss of CXCR6 coreceptor usage characterizes pathogenic lentiviruses.
Wetzel KS, Yi Y, Yadav A, Bauer AM, Bello EA, Romero DC, Bibollet-Ruche F, Hahn BH, Paiardini M, Silvestri G, Peeters M, Collman RG. Wetzel KS, et al. Among authors: bello ea. PLoS Pathog. 2018 Apr 16;14(4):e1007003. doi: 10.1371/journal.ppat.1007003. eCollection 2018 Apr. PLoS Pathog. 2018. PMID: 29659623 Free PMC article.