Gene therapy for inherited immunodeficiency. (June 2014)

• Record Type: Journal Article
• Authors: Touzot, Fabien; Hacein-Bey-Abina, Salima; Fischer, Alain; Cavazzana, Marina
• Is Part Of: Expert opinion on biological therapy. Volume 14:Number 6(2014:Jun.)
• Page Start: 789
• Page End: 798
• Publication Date: 2014-06
• Subjects: Gene therapy -- Periodicals; Protein drugs -- Periodicals; Peptide drugs -- Periodicals; Immunotherapy -- Periodicals; Drug delivery systems -- Periodicals; 615.5
• DOI: 10.1517/14712598.2014.895811
• Languages: English
• ISSNs: 1471-2598

Abstract

<abstract> <title> <x xml:space="preserve">Abstract</x> </title> <p> <bold> <italic>Introduction:</italic> </bold> During the last decade, gene therapy has emerged as a convincing therapy for primary immunodeficiencies (PIDs). <italic>Ex vivo</italic> gene transfer into autologous hematopoietic stem cells (HSCs) via viral vectors permits sustained correction of T cell immunodeficiency in two forms of severe combined immunodeficiency: X–linked SCID (SCID-X1) (γ chain [γc] deficiency) and adenosine deaminase deficiency. However, this success has been balanced by the occurrence of genotoxicity generated by the integration of first-generation retroviral vectors. Recently, the development of safer self-inactivating vectors has led to the initiation of new studies with the hope of equivalent efficacy and a better safety profile.</p> <p> <bold> <italic>Areas covered:</italic> </bold> This review article focuses on the updated results of gene therapy trials for PIDs – from early studies to ongoing clinical trials. We detail the major advances made in gene transfer and repair technologies, and discuss the many ways to extend our present experience.</p> <p> <bold> <italic>Expert opinion:</italic> </bold> With optimization in terms of safety and efficacy, gene therapy by lentiviral transduction could become a compelling alternative to allogeneic HSC transplantation, and thus may take center stage in the management of PIDs in coming years.</p> </abstract>