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Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
Weber L, Frati G, Felix T, Hardouin G, Casini A, Wollenschlaeger C, Meneghini V, Masson C, De Cian A, Chalumeau A, Mavilio F, Amendola M, Andre-Schmutz I, Cereseto A, El Nemer W, Concordet JP, Giovannangeli C, Cavazzana M, Miccio A. Weber L, et al. Among authors: el nemer w. Sci Adv. 2020 Feb 12;6(7):eaay9392. doi: 10.1126/sciadv.aay9392. Print 2020 Feb. Sci Adv. 2020. PMID: 32917636 Free PMC article.
Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M. Ribeil JA, et al. Among authors: el nemer w. N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677. N Engl J Med. 2017. PMID: 28249145 Free article.
Glycophorin-C sialylation regulates Lu/BCAM adhesive capacity during erythrocyte aging.
Klei TRL, de Back DZ, Asif PJ, Verkuijlen PJJH, Veldthuis M, Ligthart PC, Berghuis J, Clifford E, Beuger BM, van den Berg TK, van Zwieten R, El Nemer W, van Bruggen R. Klei TRL, et al. Among authors: el nemer w. Blood Adv. 2018 Jan 3;2(1):14-24. doi: 10.1182/bloodadvances.2017013094. eCollection 2018 Jan 9. Blood Adv. 2018. PMID: 29344581 Free PMC article.
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.
Antoniani C, Meneghini V, Lattanzi A, Felix T, Romano O, Magrin E, Weber L, Pavani G, El Hoss S, Kurita R, Nakamura Y, Cradick TJ, Lundberg AS, Porteus M, Amendola M, El Nemer W, Cavazzana M, Mavilio F, Miccio A. Antoniani C, et al. Among authors: el hoss s, el nemer w. Blood. 2018 Apr 26;131(17):1960-1973. doi: 10.1182/blood-2017-10-811505. Epub 2018 Mar 8. Blood. 2018. PMID: 29519807 Free article.
Prognostic factors of disease severity in infants with sickle cell anemia: A comprehensive longitudinal cohort study.
Brousse V, El Hoss S, Bouazza N, Arnaud C, Bernaudin F, Pellegrino B, Guitton C, Odièvre-Montanié MH, Mames D, Brouzes C, Picard V, Nguyen-Khoa T, Pereira C, Lapouméroulie C, Pissard S, Gardner K, Menzel S, Le Van Kim C, Colin-Aronovicz Y, Buffet P, Mohandas N, Elie C, Maier-Redelsperger M, El Nemer W, de Montalembert M. Brousse V, et al. Among authors: el hoss s, el nemer w. Am J Hematol. 2018 Nov;93(11):1411-1419. doi: 10.1002/ajh.25260. Epub 2018 Sep 21. Am J Hematol. 2018. PMID: 30132969 Free article. Clinical Trial.
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.
Weber L, Poletti V, Magrin E, Antoniani C, Martin S, Bayard C, Sadek H, Felix T, Meneghini V, Antoniou MN, El-Nemer W, Mavilio F, Cavazzana M, Andre-Schmutz I, Miccio A. Weber L, et al. Among authors: el nemer w. Mol Ther Methods Clin Dev. 2018 Aug 4;10:268-280. doi: 10.1016/j.omtm.2018.07.012. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30140714 Free PMC article.
A microfluidic approach to study the effect of mechanical stress on erythrocytes in sickle cell disease.
Lizarralde Iragorri MA, El Hoss S, Brousse V, Lefevre SD, Dussiot M, Xu T, Ferreira AR, Lamarre Y, Silva Pinto AC, Kashima S, Lapouméroulie C, Covas DT, Le Van Kim C, Colin Y, Elion J, Français O, Le Pioufle B, El Nemer W. Lizarralde Iragorri MA, et al. Among authors: el hoss s, el nemer w. Lab Chip. 2018 Sep 26;18(19):2975-2984. doi: 10.1039/c8lc00637g. Lab Chip. 2018. PMID: 30168832
Insights into determinants of spleen injury in sickle cell anemia.
El Hoss S, Cochet S, Marin M, Lapouméroulie C, Dussiot M, Bouazza N, Elie C, de Montalembert M, Arnaud C, Guitton C, Pellegrino B, Odièvre MH, Moati F, Le Van Kim C, Aronovicz YC, El Nemer W, Brousse V. El Hoss S, et al. Among authors: el nemer w. Blood Adv. 2019 Aug 13;3(15):2328-2336. doi: 10.1182/bloodadvances.2019000106. Blood Adv. 2019. PMID: 31391165 Free PMC article.
Fetal hemoglobin rescues ineffective erythropoiesis in sickle cell disease.
El Hoss S, Cochet S, Godard A, Yan H, Dussiot M, Frati G, Boutonnat-Faucher B, Laurance S, Renaud O, Joseph L, Miccio A, Brousse V, Narla M, El Nemer W. El Hoss S, et al. Among authors: el nemer w. Haematologica. 2021 Oct 1;106(10):2707-2719. doi: 10.3324/haematol.2020.265462. Haematologica. 2021. PMID: 32855279 Free PMC article.
84 results