Gene therapy for the treatment of adenosine deaminase-deficient severe combined immune deficiency

Détails

ID Serval
serval:BIB_121BF6844E7B
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
Institution
Titre
Gene therapy for the treatment of adenosine deaminase-deficient severe combined immune deficiency
Périodique
Expert Opinion on Orphan Drugs
Auteur(s)
Sokolic Robert, Candotti Fabio
ISSN
2167-8707
Statut éditorial
Publié
Date de publication
22/05/2017
Peer-reviewed
Oui
Volume
5
Numéro
6
Pages
477-485
Langue
anglais
Résumé
Introduction: Adenosine deaminase (ADA) deficiency was the first human disease treated with gene therapy. Initial trials established proofs of concepts, but did not lead to cure of the severe combined immune deficiency that is the hallmark of this disease. A breakthrough trial combined gammaretroviral gene therapy with non-myeloablative conditioning of subjects unable to benefit from enzyme replacement therapy (ERT) with pegylated bovine ADA (PEG-ADA). Three additional trials have confirmed the ability of gene therapy to cure the clinical immune defect of ADA deficiency.
Areas covered: This article reviews the development of gene therapy for ADA-SCID, compares the four trials that have been reported and discusses open questions remaining in the field.
Expert opinion: Gene therapy may be regarded as standard of care for the majority of patients with ADA deficiency, along with allogeneic hematopoietic cell transplantation (HCT) or ERT. The appropriate sequencing of these treatments remains uncertain. While gammaretroviral gene therapy has been approved in Europe, remaining concerns about potential genotoxicity have led to further development, including the use of lentivirus vectors and modification of peri-transplant management. The ability of gene therapy to completely correct both the immunologic and non-immunologic manifestations of ADA deficiency remains to be demonstrated.
Création de la notice
23/10/2017 11:50
Dernière modification de la notice
20/08/2019 12:39
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