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Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Mamcarz E, et al. Among authors: malech hl. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. N Engl J Med. 2019. PMID: 30995372 Free PMC article. Clinical Trial.
Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.
Avilés Mendoza GJ, Seidel NE, Otsu M, Anderson SM, Simon-Stoos K, Herrera A, Hoogstraten-Miller S, Malech HL, Candotti F, Puck JM, Bodine DM. Avilés Mendoza GJ, et al. Among authors: malech hl. Mol Ther. 2001 Apr;3(4):565-73. doi: 10.1006/mthe.2001.0292. Mol Ther. 2001. PMID: 11319919 Free article.
Sarcoidosis in chronic granulomatous disease.
De Ravin SS, Naumann N, Robinson MR, Barron KS, Kleiner DE, Ulrick J, Friend J, Anderson VL, Darnell D, Kang EM, Malech HL. De Ravin SS, et al. Among authors: malech hl. Pediatrics. 2006 Mar;117(3):e590-5. doi: 10.1542/peds.2005-1349. Epub 2006 Feb 1. Pediatrics. 2006. PMID: 16452321
375 results