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T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.
Kohn DB, Hershfield MS, Carbonaro D, Shigeoka A, Brooks J, Smogorzewska EM, Barsky LW, Chan R, Burotto F, Annett G, Nolta JA, Crooks G, Kapoor N, Elder M, Wara D, Bowen T, Madsen E, Snyder FF, Bastian J, Muul L, Blaese RM, Weinberg K, Parkman R. Kohn DB, et al. Among authors: wara d. Nat Med. 1998 Jul;4(7):775-80. doi: 10.1038/nm0798-775. Nat Med. 1998. PMID: 9662367 Free PMC article.
Amendment to Clinical Research Project. Project 90-C-195. April 1, 1993. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with autologous lymphocytes transduced with a human ADA gene.
Dunbar C, Chang L, Mullen C, Ramsey WJ, Carter C, Kohn D, Parkman R, Lenarsky C, Weinberg K, Wara D, Culver KW, Anderson WF, Leitman S, Fleisher T, Klein H, Shearer G, Clerici M, McGarrity G, Bastian J, Hershfield MS. Dunbar C, et al. Among authors: wara d. Hum Gene Ther. 1999 Feb 10;10(3):477-88. doi: 10.1089/10430349950018913. Hum Gene Ther. 1999. PMID: 10048399 No abstract available.
A novel X-linked disorder of immune deficiency and hypohidrotic ectodermal dysplasia is allelic to incontinentia pigmenti and due to mutations in IKK-gamma (NEMO).
Zonana J, Elder ME, Schneider LC, Orlow SJ, Moss C, Golabi M, Shapira SK, Farndon PA, Wara DW, Emmal SA, Ferguson BM. Zonana J, et al. Among authors: wara dw. Am J Hum Genet. 2000 Dec;67(6):1555-62. doi: 10.1086/316914. Epub 2000 Oct 24. Am J Hum Genet. 2000. PMID: 11047757 Free PMC article.
212 results