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Titolo:
Gene therapy of severe combined immunodeficiencies
Autore:
Fischer, A; Hacein-Bey, S; Le Deist, F; Soudais, C; Di Santo, JP; Basile, GD; Cavazzana-Calvo, M;
Indirizzi:
Hop Necker Enfants Malad, INSERM, U429, F-75730 Paris 15, France Hop Necker Enfants Malad Paris France 15 U429, F-75730 Paris 15, France Inst Pasteur, Dept Immunol, Unite Cytokines & Dev Lymphoide, F-75724 Paris, France Inst Pasteur Paris France F-75724 & Dev Lymphoide, F-75724 Paris, France
Titolo Testata:
IMMUNOLOGICAL REVIEWS
, volume: 178, anno: 2000,
pagine: 13 - 20
SICI:
0105-2896(200012)178:<13:GTOSCI>2.0.ZU;2-F
Fonte:
ISI
Lingua:
ENG
Soggetto:
BONE-MARROW TRANSPLANTATION; CLASS-II DEFICIENCY; ADENOSINE-DEAMINASE DEFICIENCY; BARE LYMPHOCYTE SYNDROME; HEMATOPOIETIC PROGENITOR CELLS; RECEPTOR-GAMMA CHAIN; IN-VIVO; RETROVIRAL VECTORS; STABLE TRANSDUCTION; LENTIVIRAL VECTOR;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
61
Recensione:
Indirizzi per estratti:
Indirizzo: Fischer, A Hop Necker Enfants Malad, INSERM, U429, 149 Rue Sevres, F-75730Paris 15, France Hop Necker Enfants Malad 149 Rue Sevres Paris France 15 France
Citazione:
A. Fischer et al., "Gene therapy of severe combined immunodeficiencies", IMMUNOL REV, 178, 2000, pp. 13-20

Abstract

Primary immunodeficiency diseases (PID) are attractive candidates for a gene therapy approach because many of these disorders convey a poor prognosiswhile a number of the genes mutated in these conditions have been identified. Gene transfer into hematopoietic stem cells (HSC) should, in theory, lead to a cure of the disease. There are, however, a number of limitations mostly related to the failure of clinically available vectors to enable transgene integration into HSC. Nevertheless PID due to a gene defect leading tofailure of cell development could be amenable to gene therapy given the selective advantage conferred to transgene expression in progenitor cells. Terminally differentiated cells are, however, long lived, as is the case for T lymphocytes. This concept led to the first gene therapy trials for adenosine deaminase (ADA) deficiency several years ago. Results were in part disappointing mostly because of the concomitant substitutive treatment by polyethylene glycol-ADA. However, recent application to X-linked severe combinedimmunodeficiency (gamma (c) deficiency) turned out to be efficient at least on a relatively short term basis (i.e. one year so far). These results demonstrate that this concept is valid and can be the basis for the treatmentof other forms of severe T-cell immunodeficiencies. Obviously, developmentof vectors (lentiviruses) able to efficiently target HSC could in the future considerably enlarge the field of PID treatable by gene transfer.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 07/08/20 alle ore 00:40:24