dc.contributor.author | Jordan, Bertrand | - |
dc.date.accessioned | 2019-11-05T12:41:10Z | |
dc.date.available | 2019-11-05T12:41:10Z | |
dc.date.issued | 2018 | |
dc.identifier.citation | Jordan, Bertrand ; Hémophilie : la thérapie génique, enfin… : Chroniques Génomiques, Med Sci (Paris), , Vol. 34, N° 3 ; p. 267-274 ; DOI : 10.1051/medsci/20183403016 | |
dc.identifier.issn | 1958-5381 | |
dc.identifier.uri | http://hdl.handle.net/10608/9758 | |
dc.description.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. | en |
dc.language.iso | fr | |
dc.publisher | EDP Sciences | |
dc.relation.ispartof | Forum | |
dc.rights | Article en libre accès | fr |
dc.rights | Médecine/Sciences - Inserm - SRMS | fr |
dc.source | M/S. Médecine sciences [ISSN papier : 0767-0974 ; ISSN numérique : 1958-5381], , Vol. 34, N° 3; p. 267-274 | |
dc.subject.mesh | Animaux | fr |
dc.subject.mesh | Dependovirus | fr |
dc.subject.mesh | Techniques de transfert de gènes | fr |
dc.subject.mesh | Thérapie génétique | fr |
dc.subject.mesh | Vecteurs génétiques | fr |
dc.subject.mesh | Hémophilie A | fr |
dc.subject.mesh | Histoire du 20ème siècle | fr |
dc.subject.mesh | Histoire du 21ème siècle | fr |
dc.subject.mesh | Humains | fr |
dc.subject.mesh | Résultat thérapeutique | fr |
dc.subject.mesh | génétique | fr |
dc.subject.mesh | histoire | fr |
dc.subject.mesh | tendances | fr |
dc.subject.mesh | méthodes | fr |
dc.subject.mesh | thérapie | fr |
dc.title | Hémophilie : la thérapie génique, enfin… : Chroniques Génomiques | fr |
dc.title.alternative | Effective gene therapy for hemophilia, at last… | en |
dc.type | Article | |
dc.contributor.affiliation | UMR 7268 ADÉS, Aix-Marseille, Université/EFS/CNRS ; CoReBio PACA, case 901, Parc scientifique de Luminy, 13288 Marseille Cedex 09, France | |
dc.identifier.doi | 10.1051/medsci/20183403016 | |
dc.identifier.pmid | 29547114 | |