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

Détails

ID Serval
serval:BIB_39E9AB4FA040
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Synthèse (review): revue aussi complète que possible des connaissances sur un sujet, rédigée à partir de l'analyse exhaustive des travaux publiés.
Collection
Publications
Titre
Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials
Périodique
Acta Haematol
Auteur⸱e⸱s
Onodera M., Nelson D. M., Sakiyama Y., Candotti F., Blaese R. M.
ISSN
0001-5792 (Print)
ISSN-L
0001-5792
Statut éditorial
Publié
Date de publication
1999
Volume
101
Numéro
2
Pages
89-96
Langue
anglais
Notes
Onodera, M
Nelson, D M
Sakiyama, Y
Candotti, F
Blaese, R M
eng
Review
Switzerland
Acta Haematol. 1999;101(2):89-96.
Résumé
Severe combined immunodeficiency (SCID) caused by adenosine deaminase deficiency (ADA-) is the first genetic disorder to be treated with gene therapy. Since 1990 when the first trial started for 2 patients with ADA- SCID, five clinical trials enrolling 11 patients have been conducted with different clinical approaches and the results obtained from these trials have recently been reported. According to these reports, T cell-directed gene transfer was useful in the treatment of ADA- SCID whereas the retroviral-mediated gene transfer to hematopoietic stem cells was insufficient for achievement of clinical benefits. This chapter reviews several crucial problems inherent in the current retroviral technology based on the clinical data observed in these pioneering ADA gene therapy trials and presents our new retroviral vector system for the next stem cell gene therapy.
Mots-clé
Adenosine Deaminase/*deficiency/genetics, Animals, Clinical Trials as Topic, *Genetic Therapy, *Genetic Vectors, Humans, Retroviridae/*genetics, Severe Combined Immunodeficiency/enzymology/*therapy
Pubmed
Création de la notice
01/11/2017 10:29
Dernière modification de la notice
20/08/2019 13:29
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