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Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair.
Perez-Bermejo JA, Efagene O, Matern WM, Holden JK, Kabir S, Chew GM, Andreoletti G, Catton E, Ennis CL, Garcia A, Gerstenberg TL, Hill KA, Jain A, Krassovsky K, Lalisan CD, Lord D, Quejarro BJ, Sales-Lee J, Shah M, Silva BJ, Skowronski J, Strukov YG, Thomas J, Veraz M, Vijay T, Wallace KA, Yuan Y, Grogan JL, Wienert B, Lahiri P, Treusch S, Dever DP, Soros VB, Partridge JR, Seim KL. Perez-Bermejo JA, et al. Among authors: dever dp. Nat Commun. 2024 Mar 23;15(1):2625. doi: 10.1038/s41467-024-46816-5. Nat Commun. 2024. PMID: 38521763 Free PMC article.
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH. Lattanzi A, et al. Among authors: dever dp. Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444. Sci Transl Med. 2021. PMID: 34135108 Free PMC article.
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.
Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI, Porteus MH. Dever DP, et al. Nature. 2016 Nov 17;539(7629):384-389. doi: 10.1038/nature20134. Epub 2016 Nov 7. Nature. 2016. PMID: 27820943 Free PMC article.
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells.
Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, Bode NM, McNeill MS, Yan S, Camarena J, Lee CM, Park SH, Wiebking V, Bak RO, Gomez-Ospina N, Pavel-Dinu M, Sun W, Bao G, Porteus MH, Behlke MA. Vakulskas CA, et al. Among authors: dever dp. Nat Med. 2018 Aug;24(8):1216-1224. doi: 10.1038/s41591-018-0137-0. Epub 2018 Aug 6. Nat Med. 2018. PMID: 30082871 Free PMC article.
Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase.
Wilkes MC, Siva K, Chen J, Varetti G, Youn MY, Chae H, Ek F, Olsson R, Lundbäck T, Dever DP, Nishimura T, Narla A, Glader B, Nakauchi H, Porteus MH, Repellin CE, Gazda HT, Lin S, Serrano M, Flygare J, Sakamoto KM. Wilkes MC, et al. Among authors: dever dp. Nat Commun. 2020 Jul 3;11(1):3344. doi: 10.1038/s41467-020-17100-z. Nat Commun. 2020. PMID: 32620751 Free PMC article.
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.
Cromer MK, Camarena J, Martin RM, Lesch BJ, Vakulskas CA, Bode NM, Kurgan G, Collingwood MA, Rettig GR, Behlke MA, Lemgart VT, Zhang Y, Goyal A, Zhao F, Ponce E, Srifa W, Bak RO, Uchida N, Majeti R, Sheehan VA, Tisdale JF, Dever DP, Porteus MH. Cromer MK, et al. Among authors: dever dp. Nat Med. 2021 Apr;27(4):677-687. doi: 10.1038/s41591-021-01284-y. Epub 2021 Mar 18. Nat Med. 2021. PMID: 33737751 Free PMC article.
32 results