Riassunto
Questo capitolo descrive in maniera analitica i diversi tipi di molecole di cui la terapia genica fa uso. Queste possono essere schematicamente divise in due categorie: 1) sequenze di DNA che contengono l’informazione per sintetizzare proteine, le quali possono svolgere funzioni diverse; 2) acidi nucleici (DNA o RNA) con funzione regolatoria, utilizzabili come piccole molecole di sintesi o, nel caso degli RNA, esprimibili all’interno delle cellule dopo il trasferimento dei relativi geni. La Tabella 2.1 riporta un elenco dei diversi acidi nucleici con possibile funzione terapeutica.
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Giacca, M. (2011). Acidi nucleici con funzione terapeutica. In: Terapia Genica. Springer, Milano. https://doi.org/10.1007/978-88-470-1989-8_2
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DOI: https://doi.org/10.1007/978-88-470-1989-8_2
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