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Titolo:
Gene therapy of primary immunodeficiencies: experimental approach and preliminary clinical results
Autore:
Fischer, B; de Saint-Basile, G; Hacein-Bey, S; Soudais, C; Di Santo, J; Cavazzana-Calvo, M;
Indirizzi:
Hop Necker Enfants Malad, INSERM, U429, F-75743 Paris 15, France Hop Necker Enfants Malad Paris France 15 U429, F-75743 Paris 15, France
Titolo Testata:
M S-MEDECINE SCIENCES
fascicolo: 5, volume: 15, anno: 1999,
pagine: 606 - 614
SICI:
0767-0974(199905)15:5<606:GTOPIE>2.0.ZU;2-O
Fonte:
ISI
Lingua:
FRE
Soggetto:
ADENOSINE-DEAMINASE DEFICIENCY; BLOOD CD34(+) CELLS; BONE-MARROW; LYMPHOCYTE DEVELOPMENT; NONDIVIDING CELLS; MAMMALIAN-CELLS; T-LYMPHOCYTES; TRANSDUCTION; EXPRESSION; DATABASE;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
51
Recensione:
Indirizzi per estratti:
Indirizzo: Fischer, B Hopranceer Enfants Malad, INSERM, U429, 149 Rue Sevres, F-75743Paris 15, F Hop Necker Enfants Malad 149 Rue Sevres Paris France 15 15, F
Citazione:
B. Fischer et al., "Gene therapy of primary immunodeficiencies: experimental approach and preliminary clinical results", M S-MED SCI, 15(5), 1999, pp. 606-614

Abstract

Primary immunodeficiencies have been long considered as a possible experimental field for gene therapy. Adenosine deaminase (ADA) deficiency was the first inherited disease for which clinical gene therapy was performed. Infusion of T cells in which the ADA gene had ben retrovirally transfered, led to sustained detection of transduced and functional T cells at least in 2 cases. ADA gene transfer into CD34 hematopoietic precursor cells was less successful although detection of transduced T cells 4 years after gene transfer has been reported. The low number of transduced T cells, however, was not sufficient to provide clinical benefit. Many factors could have influenced the outcome of these clinical studies, including partial loss of expectedselective advantage caused by concomitant PEG-ADA therapy and, obviously, limitations of presently available murine-derived retroviral vectors to transduce human cells. Possible applications of gene transfer to the treatmentof other forms of SCID including gc and JAK-3 deficiency are herein discussed based on encouraging in vitro and in vivo experimental results. It is likely that advances in vector technology and/or hematopoietic stem cell manipulation will be required to improve transduction efficiencies in hopes ofachieving significant clinical benefits of gene therapy for the many primary immunodeficiencies. This will be particularly important for situations in which transgene expression will not confer a selective growth on survivaladvantage, as for instance in functional deficiencies in phagocytic cells.

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Documento generato il 06/07/20 alle ore 07:51:14