Recent advances in gene therapy for severe congenital immunodeficiency diseases

Détails

ID Serval
serval:BIB_39E5BDCC18B8
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
Recent advances in gene therapy for severe congenital immunodeficiency diseases
Périodique
Curr Opin Hematol
Auteur(s)
Sokolic R., Kesserwan C., Candotti F.
ISSN
1531-7048 (Electronic)
ISSN-L
1065-6251
Statut éditorial
Publié
Date de publication
07/2008
Volume
15
Numéro
4
Pages
375-80
Langue
anglais
Notes
Sokolic, Robert
Kesserwan, Chimene
Candotti, Fabio
eng
Z01 HG000121-10/Intramural NIH HHS/
Z01 HG000122-10/Intramural NIH HHS/
Z99 HG999999/Intramural NIH HHS/
Research Support, N.I.H., Intramural
Review
Curr Opin Hematol. 2008 Jul;15(4):375-80. doi: 10.1097/MOH.0b013e328302c807.
Résumé
PURPOSE OF REVIEW: To discuss new data on the safety and efficacy of the ongoing gene therapy trials for primary immune deficiencies, the first reports of new trials and the preclinical developments that are likely to be translated to the clinic in the near future. RECENT FINDINGS: Both clinical successes and severe adverse events continue to be reported in trials of gammaretroviral gene therapy for severe combined immune deficiency-X1, adenosine deaminase-deficient forms of severe combined immune deficiency and chronic granulomatous disease. Insertion site analyses of recently reported trials on all of these diseases have discovered preferential insertion in the 5' ends of genes, including potentially dangerous ones such as proto-oncogenes and signal transduction and proliferation genes. Preclinical work on rodent and canine models has tested novel vectors, including lentiviruses and foamy viruses. SUMMARY: Gene therapy for the most common forms of severe combined immune deficiency can lead to immune reconstitution in most patients, although a minority of patients has derived minimal clinical benefit and some have suffered severe adverse events including death. Ongoing preclinical work attempts to address the latter shortcoming. Meanwhile, in the presence of a careful risk-benefit assessment, gene therapy remains an appropriate subject of clinical investigation.
Mots-clé
*Genetic Therapy, Humans, Immune System/physiology, Regeneration, Severe Combined Immunodeficiency/*therapy
Pubmed
Création de la notice
01/11/2017 11:29
Dernière modification de la notice
20/08/2019 14:29
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