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Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

Gene and cell therapy research recently reached a fundamental milestone toward the goal to deliver new medicines for orphan diseases. In 2016, the European Commission granted market approval to GlaxoSmithKline (GSK) for ex vivo hematopoietic stem cell (HSC) gene therapy for the treatment of adenosin...

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Détails bibliographiques
Publié dans:EMBO Mol Med
Auteurs principaux: Aiuti, Alessandro, Roncarolo, Maria Grazia, Naldini, Luigi
Format: Artigo
Langue:Inglês
Publié: John Wiley and Sons Inc. 2017
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5452047/
https://ncbi.nlm.nih.gov/pubmed/28396566
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201707573
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