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Sperimentazioni cliniche di terapia genica

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A partire dal 1989, anno della prima applicazione della terapia genica nei pazienti (vedi sezione su Geni come farmaci), sono state eseguite più di 1600 sperimentazioni cliniche, che hanno coinvolto alcune decine di migliaia di pazienti. Se valutato in termini di reale effetto terapeutico conseguito, il successo complessivo di queste sperimentazioni è stato obiettivamente modesto. Salvo alcune eclatanti eccezioni, la maggior parte delle sperimentazioni si è scontrata con problematiche di ordine sia tecnologico sia biologico probabilmente superiori a quelle attese. Tuttavia, a distanza di oltre 20 anni dalla prima applicazione, le prospettive di successo a breve termine della terapia genica oggi appaiono sicuramente migliori. Infatti da un lato i progressi compiuti in termini di veicolazione sempre più efficiente degli acidi nucleici in vivo ed ex vivo e dall’altro l’identificazione di nuovi geni con potenzialità terapeutica consentono di essere estremamente fiduciosi nel successo finale della terapia genica quale strumento per il trattamento efficace di svariate patologie umane.

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  1. Centro Internazionale di Ingegneria Genetica e Biotecnologia (ICGEB), Trieste, Italia

    Mauro Giacca

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  1. Mauro Giacca
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Giacca, M. (2011). Sperimentazioni cliniche di terapia genica. In: Terapia Genica. Springer, Milano. https://doi.org/10.1007/978-88-470-1989-8_4

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