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Titolo:
Gene-transferred oligoclonal T cells predominantly persist in peripheral blood from an adenosine deaminase-deficient patient during gene therapy
Autore:
Misaki, Y; Ezaki, I; Ariga, T; Kawamura, N; Sakiyama, Y; Yamamoto, K;
Indirizzi:
Univ Tokyo, Dept Allergy & Rheumatol, Grad Sch Med, Bunkyo Ku, Tokyo 1138655, Japan Univ Tokyo Tokyo Japan 1138655 Sch Med, Bunkyo Ku, Tokyo 1138655, Japan Kyushu Univ, Med Inst Bioregulat, Dept Clin Immunol, Beppu, Oita 87408838,Japan Kyushu Univ Beppu Oita Japan 87408838 Immunol, Beppu, Oita 87408838,Japan Hokkaido Univ, Sch Med, Dept Pediat, Sapporo, Hokkaido 0608638, Japan Hokkaido Univ Sapporo Hokkaido Japan 0608638 oro, Hokkaido 0608638, Japan
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 1, volume: 3, anno: 2001,
pagine: 24 - 27
SICI:
1525-0016(200101)3:1<24:GOTCPP>2.0.ZU;2-W
Fonte:
ISI
Lingua:
ENG
Soggetto:
IN-VIVO; RHEUMATOID-ARTHRITIS; BONE-MARROW; CLONOTYPES; EXPANSION;
Keywords:
adenosine deaminase; ADA deficiency; severe combined immunodeficiency disease; gene therapy; retroviral vector; T cell clonotype; T cell receptor-specific RT-PCR/SSCP; oligoclonality; V beta; G418 selection;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
18
Recensione:
Indirizzi per estratti:
Indirizzo: Misaki, Y Univ Tokyo, Dept Allergy & Rheumatol, Grad Sch Med, Bunkyo Ku, 7-3-1 Hongo, Tokyo 1138655, Japan Univ Tokyo 7-3-1 Hongo Tokyo Japan 1138655 Tokyo 1138655, Japan
Citazione:
Y. Misaki et al., "Gene-transferred oligoclonal T cells predominantly persist in peripheral blood from an adenosine deaminase-deficient patient during gene therapy", MOL THER, 3(1), 2001, pp. 24-27

Abstract

Adenosine deaminase (ADA) deficiency is the primary cause of severe combined immunodeficiency disease and has become a focus for developing innovative approaches to gene therapy. We previously described successful treatment of a Japanese ADA-deficient patient by periodic infusions of genetically modified autologous T lymphocytes transduced with a retroviral vector containing human ADA cDNA. In order to investigate whether polyclonality was restored by the gene therapy and whether the gene-transduced T lymphocytes persisted in the peripheral blood of the patient, we analyzed the T cell clonotype using a T cell receptor-specific RT-PCR/SSCP method. Oligoclonal T cell expansion was observed in every V beta family, and the expanded T cell clones were stable throughout the periodic gene therapy. Some of these T cell clones are likely carrying the vector, since they were identical to the clones selected by G418 resistance. Therefore, although it is uncertain when oligoclonal T cells started to expand and what percentage of the oligoclones carries the vector, the peripheral blood of the patient administered the gene therapy included oligoclonal T cells, some of which were identical to the ADA-gene-transduced clones.

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Documento generato il 04/04/20 alle ore 15:17:31