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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: srifa w. Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444. Sci Transl Med. 2021. PMID: 34135108 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: srifa w. 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.
Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.
Bonafont J, Mencía A, Chacón-Solano E, Srifa W, Vaidyanathan S, Romano R, Garcia M, Hervás-Salcedo R, Ugalde L, Duarte B, Porteus MH, Del Rio M, Larcher F, Murillas R. Bonafont J, et al. Among authors: srifa w. Mol Ther. 2021 Jun 2;29(6):2008-2018. doi: 10.1016/j.ymthe.2021.02.019. Epub 2021 Feb 18. Mol Ther. 2021. PMID: 33609734 Free PMC article.
Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: srifa w. Nat Commun. 2019 Dec 4;10(1):5624. doi: 10.1038/s41467-019-13620-5. Nat Commun. 2019. PMID: 31796738 Free PMC article.
Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: srifa w. Nat Commun. 2019 Apr 26;10(1):2021. doi: 10.1038/s41467-019-10080-9. Nat Commun. 2019. PMID: 31028274 Free PMC article.
Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: srifa w. Nat Commun. 2019 Apr 9;10(1):1634. doi: 10.1038/s41467-019-09614-y. Nat Commun. 2019. PMID: 30967552 Free PMC article.
12 results