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21 results

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Page 1
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
Candotti F, Shaw KL, Muul L, Carbonaro D, Sokolic R, Choi C, Schurman SH, Garabedian E, Kesserwan C, Jagadeesh GJ, Fu PY, Gschweng E, Cooper A, Tisdale JF, Weinberg KI, Crooks GM, Kapoor N, Shah A, Abdel-Azim H, Yu XJ, Smogorzewska M, Wayne AS, Rosenblatt HM, Davis CM, Hanson C, Rishi RG, Wang X, Gjertson D, Yang OO, Balamurugan A, Bauer G, Ireland JA, Engel BC, Podsakoff GM, Hershfield MS, Blaese RM, Parkman R, Kohn DB. Candotti F, et al. Blood. 2012 Nov 1;120(18):3635-46. doi: 10.1182/blood-2012-02-400937. Epub 2012 Sep 11. Blood. 2012. PMID: 22968453 Free PMC article. Clinical Trial.
Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report.
Engel BC, Podsakoff GM, Ireland JL, Smogorzewska EM, Carbonaro DA, Wilson K, Shah A, Kapoor N, Sweeney M, Borchert M, Crooks GM, Weinberg KI, Parkman R, Rosenblatt HM, Wu SQ, Hershfield MS, Candotti F, Kohn DB. Engel BC, et al. Among authors: carbonaro da. Blood. 2007 Jan 15;109(2):503-6. doi: 10.1182/blood-2006-06-031476. Epub 2006 Sep 14. Blood. 2007. PMID: 16973956 Free PMC article.
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.
Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. Carbonaro DA, et al. Mol Ther. 2014 Mar;22(3):607-622. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20. Mol Ther. 2014. PMID: 24256635 Free PMC article.
Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency.
Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta K, Rothe M, Schambach A, Kohn DB, Malech HL, McIvor RS, Puck JM, Cowan MJ. Punwani D, et al. Among authors: carbonaro da. Hum Gene Ther. 2017 Jan;28(1):112-124. doi: 10.1089/hum.2016.064. Epub 2016 Sep 7. Hum Gene Ther. 2017. PMID: 27611239 Free PMC article.
A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodeficiency virus-1-infected children.
Kohn DB, Bauer G, Rice CR, Rothschild JC, Carbonaro DA, Valdez P, Hao Ql, Zhou C, Bahner I, Kearns K, Brody K, Fox S, Haden E, Wilson K, Salata C, Dolan C, Wetter C, Aguilar-Cordova E, Church J. Kohn DB, et al. Among authors: carbonaro da. Blood. 1999 Jul 1;94(1):368-71. Blood. 1999. PMID: 10381536 Free article. Clinical Trial.
Gene therapy for pediatric AIDS.
Bauer G, Selander D, Engel B, Carbonaro D, Csik S, Rawlings S, Church J, Kohn DB. Bauer G, et al. Ann N Y Acad Sci. 2000 Nov;918:318-29. doi: 10.1111/j.1749-6632.2000.tb05501.x. Ann N Y Acad Sci. 2000. PMID: 11131719 Review.
21 results