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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...

詳細記述

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書誌詳細
出版年:EMBO Mol Med
主要な著者: Aiuti, Alessandro, Roncarolo, Maria Grazia, Naldini, Luigi
フォーマット: Artigo
言語:Inglês
出版事項: John Wiley and Sons Inc. 2017
主題:
オンライン・アクセス: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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