Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases

Détails

ID Serval
serval:BIB_884A92DF0904
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 transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases
Périodique
Int J Hematol
Auteur(s)
Candotti F.
ISSN
1865-3774 (Electronic)
ISSN-L
0925-5710
Statut éditorial
Publié
Date de publication
04/2014
Volume
99
Numéro
4
Pages
383-92
Langue
anglais
Notes
Candotti, Fabio
eng
Review
Japan
Int J Hematol. 2014 Apr;99(4):383-92. doi: 10.1007/s12185-014-1524-z. Epub 2014 Feb 1.
Résumé
Gene transfer into the hematopoietic stem cell has shown curative potential for a variety of hematological disorders. Primary immunodeficiency diseases have led to the way in this field of gene therapy as an example and a model. Clinical results from the past 15 years have shown that significant improvement and even cure can be achieved for diseases such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease and Wiskott-Aldrich syndrome. Unfortunately, with the initial clear clinical benefits, the first serious complications of gene therapy have also occurred. In a significant number of patients treated using vectors based on murine gamma-retroviruses and carrying powerful viral enhancer elements, insertional oncogenesis events have resulted in acute leukemias that, in some cases, have had fatal outcomes. These serious adverse events have sparked a revision of the assessment of risks and benefits of integrating gene transfer for hematological diseases and prompted the development and application of new generations of viral vectors with recognized superior safety characteristics. This review summarizes the clinical experience of gene therapy for primary immunodeficiencies and discusses the likely avenues of progress in the future development of this expanding field of clinical investigations.
Mots-clé
Adenosine Deaminase/deficiency/genetics, Agammaglobulinemia/genetics/therapy, Animals, Clinical Trials as Topic, *Gene Transfer Techniques, *Genetic Therapy, Granulomatous Disease, Chronic/genetics/therapy, *Hematopoietic Stem Cell Transplantation, Hematopoietic Stem Cells/*metabolism, Humans, Immunologic Deficiency Syndromes/*genetics/*therapy, Severe Combined Immunodeficiency/genetics/therapy, Wiskott-Aldrich Syndrome/genetics/therapy, X-Linked Combined Immunodeficiency Diseases/genetics/therapy
Pubmed
Open Access
Oui
Création de la notice
01/11/2017 10:29
Dernière modification de la notice
20/08/2019 14:47
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