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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: williams da. 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.
Rac GTPases in human diseases.
Pai SY, Kim C, Williams DA. Pai SY, et al. Among authors: williams da. Dis Markers. 2010;29(3-4):177-87. doi: 10.3233/DMA-2010-0738. Dis Markers. 2010. PMID: 21178276 Free PMC article. Review.
Overcoming reprogramming resistance of Fanconi anemia cells.
Müller LU, Milsom MD, Harris CE, Vyas R, Brumme KM, Parmar K, Moreau LA, Schambach A, Park IH, London WB, Strait K, Schlaeger T, Devine AL, Grassman E, D'Andrea A, Daley GQ, Williams DA. Müller LU, et al. Among authors: williams da. Blood. 2012 Jun 7;119(23):5449-57. doi: 10.1182/blood-2012-02-408674. Epub 2012 Feb 27. Blood. 2012. PMID: 22371882 Free PMC article.
Unexpected help: mTOR meets lentiviral vectors.
Brendel C, Williams DA. Brendel C, et al. Among authors: williams da. Blood. 2014 Aug 7;124(6):832-3. doi: 10.1182/blood-2014-06-582411. Blood. 2014. PMID: 25104861 Free PMC article.
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. Hacein-Bey-Abina S, et al. Among authors: williams da. N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588. N Engl J Med. 2014. PMID: 25295500 Free PMC article. Clinical Trial.
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: williams da. 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.
1,225 results