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dc.contributor.authorJordan, Bertrand-
dc.date.accessioned2019-11-05T12:41:10Z
dc.date.available2019-11-05T12:41:10Z
dc.date.issued2018
dc.identifier.citationJordan, 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.issn1958-5381
dc.identifier.urihttp://hdl.handle.net/10608/9758
dc.description.abstractRecent 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.isofr
dc.publisherEDP Sciences
dc.relation.ispartofForum
dc.rightsArticle en libre accèsfr
dc.rightsMédecine/Sciences - Inserm - SRMSfr
dc.sourceM/S. Médecine sciences [ISSN papier : 0767-0974 ; ISSN numérique : 1958-5381], , Vol. 34, N° 3; p. 267-274
dc.subject.meshAnimauxfr
dc.subject.meshDependovirusfr
dc.subject.meshTechniques de transfert de gènesfr
dc.subject.meshThérapie génétiquefr
dc.subject.meshVecteurs génétiquesfr
dc.subject.meshHémophilie Afr
dc.subject.meshHistoire du 20ème sièclefr
dc.subject.meshHistoire du 21ème sièclefr
dc.subject.meshHumainsfr
dc.subject.meshRésultat thérapeutiquefr
dc.subject.meshgénétiquefr
dc.subject.meshhistoirefr
dc.subject.meshtendancesfr
dc.subject.meshméthodesfr
dc.subject.meshthérapiefr
dc.titleHémophilie : la thérapie génique, enfin… : Chroniques Génomiquesfr
dc.title.alternativeEffective gene therapy for hemophilia, at last…en
dc.typeArticle
dc.contributor.affiliationUMR 7268 ADÉS, Aix-Marseille, Université/EFS/CNRS ; CoReBio PACA, case 901, Parc scientifique de Luminy, 13288 Marseille Cedex 09, France
dc.identifier.doi10.1051/medsci/20183403016
dc.identifier.pmid29547114


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