| Issue |
Med Sci (Paris)
Volume 34, Number 3, Mars 2018
|
|
|---|---|---|
| Page(s) | 267 - 274 | |
| Section | Forum | |
| DOI | https://doi.org/10.1051/medsci/20183403016 | |
| Published online | 16 March 2018 | |
Chroniques Génomiques
Hémophilie : la thérapie génique, enfin…
Chroniques Génomiques
Effective gene therapy for hemophilia, at last…
UMR 7268 ADÉS, Aix-Marseille, Université/EFS/CNRS ; CoReBio PACA, case 901, Parc scientifique de Luminy, 13288 Marseille Cedex 09, France
Abstract
Recent efforts at gene therapy for haemophilia A and B using AAV-derived vectors show durable expression of coagulation factors at significant levels, resulting in almost complete correction of the phenotype. This is the first success of gene therapy for a major hereditary disorder, and shows how continuous improvement of many components of the system has finally succeeded. Although these results must be confirmed with more patients and longer durations, they constitute a significant accomplishment for this approach after decades of frustration.
© 2018 médecine/sciences – Inserm
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