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22. Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug. Ferrua F; Aiuti A Hum Gene Ther; 2017 Nov; 28(11):972-981. PubMed ID: 28847159 [TBL] [Abstract][Full Text] [Related]
23. Flow cytometry analysis of adenosine deaminase (ADA) expression: a simple and reliable tool for the assessment of ADA-deficient patients before and after gene therapy. Otsu M; Hershfield MS; Tuschong LM; Muul LM; Onodera M; Ariga T; Sakiyama Y; Candotti F Hum Gene Ther; 2002 Feb; 13(3):425-32. PubMed ID: 11860709 [TBL] [Abstract][Full Text] [Related]
26. Bone marrow transplantation and alternatives for adenosine deaminase deficiency. Gaspar HB Immunol Allergy Clin North Am; 2010 May; 30(2):221-36. PubMed ID: 20493398 [TBL] [Abstract][Full Text] [Related]
27. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients. Bordignon C; Notarangelo LD; Nobili N; Ferrari G; Casorati G; Panina P; Mazzolari E; Maggioni D; Rossi C; Servida P; Ugazio AG; Mavilio F Science; 1995 Oct; 270(5235):470-5. PubMed ID: 7570000 [TBL] [Abstract][Full Text] [Related]
28. Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies. Silver JN; Flotte TR Pharmacogenomics; 2008 Jul; 9(7):947-68. PubMed ID: 18597656 [TBL] [Abstract][Full Text] [Related]
29. 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 Nat Med; 1998 Jul; 4(7):775-80. PubMed ID: 9662367 [TBL] [Abstract][Full Text] [Related]
30. The ADA human gene therapy clinical protocol. Hum Gene Ther; 1990; 1(3):327-62. PubMed ID: 2081198 [TBL] [Abstract][Full Text] [Related]
32. Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency. Reinhardt B; Habib O; Shaw KL; Garabedian E; Carbonaro-Sarracino DA; Terrazas D; Fernandez BC; De Oliveira S; Moore TB; Ikeda AK; Engel BC; Podsakoff GM; Hollis RP; Fernandes A; Jackson C; Shupien S; Mishra S; Davila A; Mottahedeh J; Vitomirov A; Meng W; Rosenfeld AM; Roche AM; Hokama P; Reddy S; Everett J; Wang X; Luning Prak ET; Cornetta K; Hershfield MS; Sokolic R; De Ravin SS; Malech HL; Bushman FD; Candotti F; Kohn DB Blood; 2021 Oct; 138(15):1304-1316. PubMed ID: 33974038 [TBL] [Abstract][Full Text] [Related]
33. In vivo kinetics of transduced cells in peripheral T cell-directed gene therapy: role of CD8+ cells in improved immunological function in an adenosine deaminase (ADA)-SCID patient. Kawamura N; Ariga T; Ohtsu M; Kobayashi I; Yamada M; Tame A; Furuta H; Okano M; Egashira M; Niikawa N; Kobayashi K; Sakiyama Y J Immunol; 1999 Aug; 163(4):2256-61. PubMed ID: 10438969 [TBL] [Abstract][Full Text] [Related]
34. The different extent of B and T cell immune reconstitution after hematopoietic stem cell transplantation and enzyme replacement therapies in SCID patients with adenosine deaminase deficiency. Serana F; Sottini A; Chiarini M; Zanotti C; Ghidini C; Lanfranchi A; Notarangelo LD; Caimi L; Imberti L J Immunol; 2010 Dec; 185(12):7713-22. PubMed ID: 21057082 [TBL] [Abstract][Full Text] [Related]
35. Gene-transferred oligoclonal T cells predominantly persist in peripheral blood from an adenosine deaminase-deficient patient during gene therapy. Misaki Y; Ezaki I; Ariga T; Kawamura N; Sakiyama Y; Yamamoto K Mol Ther; 2001 Jan; 3(1):24-7. PubMed ID: 11162307 [TBL] [Abstract][Full Text] [Related]
36. Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency. Silver JN; Elder M; Conlon T; Cruz P; Wright AJ; Srivastava A; Flotte TR Hum Gene Ther; 2011 Aug; 22(8):935-49. PubMed ID: 21142972 [TBL] [Abstract][Full Text] [Related]
37. Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells. Bodine DM; Moritz T; Donahue RE; Luskey BD; Kessler SW; Martin DI; Orkin SH; Nienhuis AW; Williams DA Blood; 1993 Oct; 82(7):1975-80. PubMed ID: 7691243 [TBL] [Abstract][Full Text] [Related]
38. Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency. Migliavacca M; Barzaghi F; Fossati C; Rancoita PMV; Gabaldo M; Dionisio F; Giannelli S; Salerio FA; Ferrua F; Tucci F; Calbi V; Gallo V; Recupero S; Consiglieri G; Pajno R; Sambuco M; Priolo A; Ferri C; Garella V; Monti I; Silvani P; Darin S; Casiraghi M; Corti A; Zancan S; Levi M; Cesana D; Carlucci F; Pituch-Noworolska A; AbdElaziz D; Baumann U; Finocchi A; Cancrini C; Ladogana S; Meinhardt A; Meyts I; Montin D; Notarangelo LD; Porta F; Pasquet M; Speckmann C; Stepensky P; Tommasini A; Rabusin M; Karakas Z; Galicchio M; Leonardi L; Duse M; Guner SN; Di Serio C; Ciceri F; Bernardo ME; Aiuti A; Cicalese MP Nat Med; 2024 Feb; 30(2):488-497. PubMed ID: 38355973 [TBL] [Abstract][Full Text] [Related]
39. Treatment of patients with severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency by autologous transplantation of genetically modified bone marrow cells. Hoogerbrugge PM; Vossen JM; v Beusechem VW; Valerio D Hum Gene Ther; 1992 Oct; 3(5):553-8. PubMed ID: 1420454 [No Abstract] [Full Text] [Related]
40. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency. Cicalese MP; Ferrua F; Castagnaro L; Pajno R; Barzaghi F; Giannelli S; Dionisio F; Brigida I; Bonopane M; Casiraghi M; Tabucchi A; Carlucci F; Grunebaum E; Adeli M; Bredius RG; Puck JM; Stepensky P; Tezcan I; Rolfe K; De Boever E; Reinhardt RR; Appleby J; Ciceri F; Roncarolo MG; Aiuti A Blood; 2016 Jul; 128(1):45-54. PubMed ID: 27129325 [TBL] [Abstract][Full Text] [Related] [Previous] [Next] [New Search]