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11. Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency. Shaw KL; Garabedian E; Mishra S; Barman P; Davila A; Carbonaro D; Shupien S; Silvin C; Geiger S; Nowicki B; Smogorzewska EM; Brown B; Wang X; de Oliveira S; Choi Y; Ikeda A; Terrazas D; Fu PY; Yu A; Fernandez BC; Cooper AR; Engel B; Podsakoff G; Balamurugan A; Anderson S; Muul L; Jagadeesh GJ; Kapoor N; Tse J; Moore TB; Purdy K; Rishi R; Mohan K; Skoda-Smith S; Buchbinder D; Abraham RS; Scharenberg A; Yang OO; Cornetta K; Gjertson D; Hershfield M; Sokolic R; Candotti F; Kohn DB J Clin Invest; 2017 May; 127(5):1689-1699. PubMed ID: 28346229 [TBL] [Abstract][Full Text] [Related]
12. [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]. Smogorzewska EM; Weinberg KI; Kohn DB Med Wieku Rozwoj; 2003; 7(1):27-34. PubMed ID: 13130167 [TBL] [Abstract][Full Text] [Related]
13. Gene therapy for adenosine deaminase deficiency. Cappelli B; Aiuti A Immunol Allergy Clin North Am; 2010 May; 30(2):249-60. PubMed ID: 20493400 [TBL] [Abstract][Full Text] [Related]
14. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. Aiuti A; Cattaneo F; Galimberti S; Benninghoff U; Cassani B; Callegaro L; Scaramuzza S; Andolfi G; Mirolo M; Brigida I; Tabucchi A; Carlucci F; Eibl M; Aker M; Slavin S; Al-Mousa H; Al Ghonaium A; Ferster A; Duppenthaler A; Notarangelo L; Wintergerst U; Buckley RH; Bregni M; Marktel S; Valsecchi MG; Rossi P; Ciceri F; Miniero R; Bordignon C; Roncarolo MG N Engl J Med; 2009 Jan; 360(5):447-58. PubMed ID: 19179314 [TBL] [Abstract][Full Text] [Related]
15. 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 Blood; 2012 Nov; 120(18):3635-46. PubMed ID: 22968453 [TBL] [Abstract][Full Text] [Related]
17. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. Kohn DB; Booth C; Shaw KL; Xu-Bayford J; Garabedian E; Trevisan V; Carbonaro-Sarracino DA; Soni K; Terrazas D; Snell K; Ikeda A; Leon-Rico D; Moore TB; Buckland KF; Shah AJ; Gilmour KC; De Oliveira S; Rivat C; Crooks GM; Izotova N; Tse J; Adams S; Shupien S; Ricketts H; Davila A; Uzowuru C; Icreverzi A; Barman P; Campo Fernandez B; Hollis RP; Coronel M; Yu A; Chun KM; Casas CE; Zhang R; Arduini S; Lynn F; Kudari M; Spezzi A; Zahn M; Heimke R; Labik I; Parrott R; Buckley RH; Reeves L; Cornetta K; Sokolic R; Hershfield M; Schmidt M; Candotti F; Malech HL; Thrasher AJ; Gaspar HB N Engl J Med; 2021 May; 384(21):2002-2013. PubMed ID: 33974366 [TBL] [Abstract][Full Text] [Related]
18. Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion. Carbonaro DA; Jin X; Cotoi D; Mi T; Yu XJ; Skelton DC; Dorey F; Kellems RE; Blackburn MR; Kohn DB Blood; 2008 Jun; 111(12):5745-54. PubMed ID: 18356486 [TBL] [Abstract][Full Text] [Related]
19. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992. Blaese RM; Culver KW; Chang L; Anderson WF; Mullen C; Nienhuis A; Carter C; Dunbar C; Leitman S; Berger M Hum Gene Ther; 1993 Aug; 4(4):521-7. PubMed ID: 7691188 [TBL] [Abstract][Full Text] [Related]
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