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Gene therapy: principles, challenges and use in clinical practice

7 May 2024

Abstract excerpt

INTRODUCTION: Gene therapy is an emerging topic in medicine. The first products have already been licensed in the European Union for the treatment of immune deficiency, spinal muscular atrophy, hemophilia, retinal dystrophy, a rare neurotransmitter disorder and some hematological cancers, while many more are being assessed in preclinical and clinical trials. OBJECTIVE: The purpose of this review is to provide an...

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PMID: 38713227
DOI: 10.1007/s00508-024-02368-8

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