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Titolo:
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
Autore:
Hacein-Bey, S; Gross, F; Nusbaum, P; Yvon, E; Fischer, A; Cavazzana-Calvo, M;
Indirizzi:
Hop Necker Enfants Malad, INSERM U429, F-75015 Paris, France Hop Necker Enfants Malad Paris France F-75015 429, F-75015 Paris, France Hop Necker Enfants Malad, ETS, F-75015 Paris, France Hop Necker Enfants Malad Paris France F-75015 ETS, F-75015 Paris, France
Titolo Testata:
PATHOLOGIE BIOLOGIE
fascicolo: 1, volume: 49, anno: 2001,
pagine: 57 - 66
SICI:
0369-8114(200102)49:1<57:GTOHSC>2.0.ZU;2-Z
Fonte:
ISI
Lingua:
FRE
Soggetto:
HEMATOPOIETIC STEM-CELLS; ADENOSINE-DEAMINASE DEFICIENCY; BONE-MARROW CELLS; RETROVIRAL VECTORS; PROGENITOR CELLS; CD34(+) CELLS; CORD-BLOOD; ORGAN-CULTURES; NOD/SCID MICE; HEMOPHILIA-B;
Keywords:
gamma c chain; gene therapy; hematopoietic progenitor cells; retroviral vector; X-linked severe combined immunodeficiency (SCID-X1);
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
48
Recensione:
Indirizzi per estratti:
Indirizzo: Hacein-Bey, S Hop Necker Enfants Malad, INSERM U429, 149 Rue Sevres, F-75015 Paris, France Hop Necker Enfants Malad 149 Rue Sevres Paris France F-75015
Citazione:
S. Hacein-Bey et al., "Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease", PATH BIOL, 49(1), 2001, pp. 57-66

Abstract

X-linked severe combined immunodeficiency (SCID-X1) is a recessive hereditary disorder in which early T and Natural Killer (NK) lymphocyte development is blocked. The genetic disorder results from mutations in the common gammac chain that participates in several cytokine receptors including the interleukin-2 (II-2), II-4, II-7, II-9 II-15 receptors. SCID-XI offers a reliable model for gene therapy as it is a lethal condition that is, in many cases, curable by allogeneic bone marrow transplantation. We have shown that retrovirus-mediated transfer of the gammac cDNA induced gammac chain expression and restored the function of the high-affinity IL-2 receptor on SCI-XI EBV-transformed B-cell lines. We have the designed culture conditions to study NK-cell and T-cell development of CD34+ hematopoietic progenitor cells.in the culture systems, gammac transduced CD34+ marrow cells from two SCID-XI patients were able to mature into CD56+ and/or CD16+ NK cells and into CD4+ TCR alpha beta+ T cells. These preclinical results set the basis for aclinical study of ex-vivo gammac gene transfer into CD34+ cells from SCID-XI patients. (C) 2001 Editions scientifiques et medicales Elsevier SAS.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 18/09/20 alle ore 10:12:00