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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. Frangoul H, et al. Among authors: lekstrom himes j. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283989 Clinical Trial.
Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del.
Taylor-Cousar JL, Munck A, McKone EF, van der Ent CK, Moeller A, Simard C, Wang LT, Ingenito EP, McKee C, Lu Y, Lekstrom-Himes J, Elborn JS. Taylor-Cousar JL, et al. Among authors: lekstrom himes j. N Engl J Med. 2017 Nov 23;377(21):2013-2023. doi: 10.1056/NEJMoa1709846. Epub 2017 Nov 3. N Engl J Med. 2017. PMID: 29099344 Free article. Clinical Trial.
Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.
Rowe SM, Daines C, Ringshausen FC, Kerem E, Wilson J, Tullis E, Nair N, Simard C, Han L, Ingenito EP, McKee C, Lekstrom-Himes J, Davies JC. Rowe SM, et al. Among authors: lekstrom himes j. N Engl J Med. 2017 Nov 23;377(21):2024-2035. doi: 10.1056/NEJMoa1709847. Epub 2017 Nov 3. N Engl J Med. 2017. PMID: 29099333 Free PMC article. Clinical Trial.
Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR.
Donaldson SH, Pilewski JM, Griese M, Cooke J, Viswanathan L, Tullis E, Davies JC, Lekstrom-Himes JA, Wang LT; VX11-661-101 Study Group. Donaldson SH, et al. Among authors: lekstrom himes ja. Am J Respir Crit Care Med. 2018 Jan 15;197(2):214-224. doi: 10.1164/rccm.201704-0717OC. Am J Respir Crit Care Med. 2018. PMID: 28930490 Free PMC article. Clinical Trial.
Immunodeficiency diseases caused by defects in phagocytes.
Lekstrom-Himes JA, Gallin JI. Lekstrom-Himes JA, et al. N Engl J Med. 2000 Dec 7;343(23):1703-14. doi: 10.1056/NEJM200012073432307. N Engl J Med. 2000. PMID: 11106721 Review. No abstract available.
39 results