Format
Sort by
Items per page

Send to

Choose Destination

Links from PubMed

Items: 1 to 20 of 121

1.

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, Greenblatt JJ, Rosenberg SA, Klein H, Berger M, Mullen CA, Ramsey WJ, Muul L, Morgan RA, Anderson WF.

Science. 1995 Oct 20;270(5235):475-80.

PMID:
7570001
2.

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 20;270(5235):470-5.

PMID:
7570000
3.

Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency.

Onodera M, Ariga T, Kawamura N, Kobayashi I, Ohtsu M, Yamada M, Tame A, Furuta H, Okano M, Matsumoto S, Kotani H, McGarrity GJ, Blaese RM, Sakiyama Y.

Blood. 1998 Jan 1;91(1):30-6.

4.

Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement.

Aiuti A, Vai S, Mortellaro A, Casorati G, Ficara F, Andolfi G, Ferrari G, Tabucchi A, Carlucci F, Ochs HD, Notarangelo LD, Roncarolo MG, Bordignon C.

Nat Med. 2002 May;8(5):423-5. No abstract available.

PMID:
11984564
5.

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 29;360(5):447-58. doi: 10.1056/NEJMoa0805817.

6.

Development of gene therapy for immunodeficiency: adenosine deaminase deficiency.

Blaese RM.

Pediatr Res. 1993 Jan;33(1 Suppl):S49-53; discussion S53-5. Review.

PMID:
8433875
7.

Retrovirus mediated gene transfer as therapy for adenosine deaminase (ADA) deficiency.

Ramsey WJ, Mullen CA, Blaese RM.

Leukemia. 1995 Oct;9 Suppl 1:S70.

PMID:
7475319
8.

Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning.

Otsu M, Yamada M, Nakajima S, Kida M, Maeyama Y, Hatano N, Toita N, Takezaki S, Okura Y, Kobayashi R, Matsumoto Y, Tatsuzawa O, Tsuchida F, Kato S, Kitagawa M, Mineno J, Hershfield MS, Bali P, Candotti F, Onodera M, Kawamura N, Sakiyama Y, Ariga T.

J Clin Immunol. 2015 May;35(4):384-98. doi: 10.1007/s10875-015-0157-1. Epub 2015 Apr 15.

PMID:
25875699
9.

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

Cassani B, Montini E, Maruggi G, Ambrosi A, Mirolo M, Selleri S, Biral E, Frugnoli I, Hernandez-Trujillo V, Di Serio C, Roncarolo MG, Naldini L, Mavilio F, Aiuti A.

Blood. 2009 Oct 22;114(17):3546-56. doi: 10.1182/blood-2009-02-202085. Epub 2009 Aug 3.

11.

Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials.

Onodera M, Nelson DM, Sakiyama Y, Candotti F, Blaese RM.

Acta Haematol. 1999;101(2):89-96. Review.

PMID:
10202239
12.

Lymphocyte gene therapy.

Culver KW, Anderson WF, Blaese RM.

Hum Gene Ther. 1991 Summer;2(2):107-9.

PMID:
1911929
13.

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 15;163(4):2256-61.

14.

Transfer of the ADA gene into bone marrow cells and peripheral blood lymphocytes for the treatment of patients affected by ADA-deficient SCID.

Bordignon C, Mavilio F, Ferrari G, Servida P, Ugazio AG, Notarangelo LD, Gilboa E, Rossini S, O'Reilly RJ, Smith CA, et al.

Hum Gene Ther. 1993 Aug;4(4):513-20.

PMID:
8399494
15.

Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning.

Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ.

Mol Ther. 2006 Oct;14(4):505-13. Epub 2006 Aug 14.

16.

[Gene therapy for adenosine deaminase deficiency].

Sakiyama Y.

Hokkaido Igaku Zasshi. 1996 Jan;71(1):27-32. Review. Japanese.

PMID:
8727372
17.
18.

Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial.

Muul LM, Tuschong LM, Soenen SL, Jagadeesh GJ, Ramsey WJ, Long Z, Carter CS, Garabedian EK, Alleyne M, Brown M, Bernstein W, Schurman SH, Fleisher TA, Leitman SF, Dunbar CE, Blaese RM, Candotti F.

Blood. 2003 Apr 1;101(7):2563-9. Epub 2002 Nov 27.

19.

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. doi: 10.1089/hum.2010.121. Epub 2011 Mar 13.

PMID:
21142972
20.

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.

Mol Ther. 2014 Mar;22(3):607-22. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20.

Supplemental Content

Support Center