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129 results

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Page 1
β-globin gene transfer to human bone marrow for sickle cell disease.
Romero Z, Urbinati F, Geiger S, Cooper AR, Wherley J, Kaufman ML, Hollis RP, de Assin RR, Senadheera S, Sahagian A, Jin X, Gellis A, Wang X, Gjertson D, Deoliveira S, Kempert P, Shupien S, Abdel-Azim H, Walters MC, Meiselman HJ, Wenby RB, Gruber T, Marder V, Coates TD, Kohn DB. Romero Z, et al. J Clin Invest. 2013 Jul 1;123(8):3317-30. doi: 10.1172/JCI67930. Online ahead of print. J Clin Invest. 2013. PMID: 23863630 Free PMC article.
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.
Human lymphoid development in the absence of common γ-chain receptor signaling.
Kohn LA, Seet CS, Scholes J, Codrea F, Chan R, Zaidi-Merchant S, Zhu Y, De Oliveira S, Kapoor N, Shah A, Abdel-Azim H, Kohn DB, Crooks GM. Kohn LA, et al. J Immunol. 2014 Jun 1;192(11):5050-8. doi: 10.4049/jimmunol.1303496. Epub 2014 Apr 25. J Immunol. 2014. PMID: 24771849 Free PMC article. Clinical Trial.
Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of clinical disease; earlier clearance of alemtuzumab may be associated with graft rejection.
Abdel-Azim H, Mahadeo KM, Zhao Q, Khazal S, Kohn DB, Crooks GM, Shah AJ, Kapoor N. Abdel-Azim H, et al. Am J Hematol. 2015 Nov;90(11):1021-6. doi: 10.1002/ajh.24141. Epub 2015 Oct 12. Am J Hematol. 2015. PMID: 26242764 Free article.
Pre- and post-natal treatment of hemophagocytic lymphohistiocytosis.
Shah AJ, Kapoor N, Cooper RM, Crooks GM, Lenarsky C, Abdel-Azim H, Wu SQ, Wilson K, Weinberg KI, Parkman R, Kohn DB. Shah AJ, et al. Pediatr Blood Cancer. 2009 Jan;52(1):139-42. doi: 10.1002/pbc.21778. Pediatr Blood Cancer. 2009. PMID: 18819128 Free PMC article.
Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases.
Arnold SD, Brazauskas R, He N, Li Y, Aplenc R, Jin Z, Hall M, Atsuta Y, Dalal J, Hahn T, Khera N, Bonfim C, Majhail NS, Diaz MA, Freytes CO, Wood WA, Savani BN, Kamble RT, Parsons S, Ahmed I, Sullivan K, Beattie S, Dandoy C, Munker R, Marino S, Bitan M, Abdel-Azim H, Aljurf M, Olsson RF, Joshi S, Buchbinder D, Eckrich MJ, Hashmi S, Lazarus H, Marks DI, Steinberg A, Saad A, Gergis U, Krishnamurti L, Abraham A, Rangarajan HG, Walters M, Lipscomb J, Saber W, Satwani P. Arnold SD, et al. Haematologica. 2017 Nov;102(11):1823-1832. doi: 10.3324/haematol.2017.169581. Epub 2017 Aug 17. Haematologica. 2017. PMID: 28818869 Free PMC article.
A reduced-toxicity regimen is associated with durable engraftment and clinical cure of nonmalignant genetic diseases among children undergoing blood and marrow transplantation with an HLA-matched related donor.
Mahadeo KM, Weinberg KI, Abdel-Azim H, Miklos DB, Killen R, Kohn D, Crooks GM, Shah AJ, Kharbanda S, Agarwal R, Kapoor N. Mahadeo KM, et al. Biol Blood Marrow Transplant. 2015 Mar;21(3):440-4. doi: 10.1016/j.bbmt.2014.11.005. Epub 2014 Nov 13. Biol Blood Marrow Transplant. 2015. PMID: 25459642 Free article. Clinical Trial.
B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation.
Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai SY. Miggelbrink AM, et al. Blood. 2018 Jun 28;131(26):2967-2977. doi: 10.1182/blood-2017-10-809822. Epub 2018 May 4. Blood. 2018. PMID: 29728406 Free PMC article. Clinical Trial.
129 results