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miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction.
Guda S, Brendel C, Renella R, Du P, Bauer DE, Canver MC, Grenier JK, Grimson AW, Kamran SC, Thornton J, de Boer H, Root DE, Milsom MD, Orkin SH, Gregory RI, Williams DA. Guda S, et al. Among authors: renella r. Mol Ther. 2015 Sep;23(9):1465-74. doi: 10.1038/mt.2015.113. Epub 2015 Jun 17. Mol Ther. 2015. PMID: 26080908 Free PMC article.
Congenital X-linked neutropenia with myelodysplasia and somatic tetraploidy due to a germline mutation in SEPT6.
Renella R, Gagne K, Beauchamp E, Fogel J, Perlov A, Sola M, Schlaeger T, Hofmann I, Shimamura A, Ebert BL, Schmitz-Abe K, Markianos K, Murphy K, Sun L, Rockowitz S, Sliz P, Campagna DR, Springer TA, Bahl C, Agarwal S, Fleming MD, Williams DA. Renella R, et al. Am J Hematol. 2022 Jan 1;97(1):18-29. doi: 10.1002/ajh.26382. Epub 2021 Nov 3. Am J Hematol. 2022. PMID: 34677878 Free PMC article.
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
Brendel C, Guda S, Renella R, Bauer DE, Canver MC, Kim YJ, Heeney MM, Klatt D, Fogel J, Milsom MD, Orkin SH, Gregory RI, Williams DA. Brendel C, et al. Among authors: renella r. J Clin Invest. 2016 Oct 3;126(10):3868-3878. doi: 10.1172/JCI87885. Epub 2016 Sep 6. J Clin Invest. 2016. PMID: 27599293 Free PMC article.
88 results