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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: london wb. 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.
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: london wb. 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.
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: london wb. 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.
Newborn Screening for Sickle Cell Disease in Liberia: A Pilot Study.
Tubman VN, Marshall R, Jallah W, Guo D, Ma C, Ohene-Frempong K, London WB, Heeney MM. Tubman VN, et al. Among authors: london wb. Pediatr Blood Cancer. 2016 Apr;63(4):671-6. doi: 10.1002/pbc.25875. Epub 2016 Jan 6. Pediatr Blood Cancer. 2016. PMID: 26739520 Free PMC article.
247 results