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Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, Armant M, Manis JP, Williams DA. Esrick EB, et al. Among authors: brendel c. N Engl J Med. 2021 Jan 21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283990 Free PMC article. Clinical Trial.
Unexpected help: mTOR meets lentiviral vectors.
Brendel C, Williams DA. Brendel C, et al. Blood. 2014 Aug 7;124(6):832-3. doi: 10.1182/blood-2014-06-582411. Blood. 2014. PMID: 25104861 Free PMC article.
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: brendel c. 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.
151 results