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EnglishOligonucleotides (ONs), the focus of the chapter herein, are mostly known for their utility to selectively manipulate RNA processing by increasing or decreasing target gene levels, in particular by inducing enzymatic RNA degradation, blocking or mimicking miRNAs, inhibiting mRNA translation or modulating pre-mRNA splicing. Since these mechanisms of action are based on the Watson–Crick base pairing to target sequences, ONs are highly specific compounds. The possibility of a large scale and standardized production of these compounds makes them attractive for the therapy of inherited disorders. To date, four ONs have received marketing authorization and more than 100 have been, or are, under clinical trials. Several different oligonucleotide chemistries have been explored, each with its own delivery hurdles and toxicology patterns. Only a limited knowledge is available concerning the cellular and subcellular mechanisms of ONs uptake, transport and metabolism, presently making the improvement of ONs’ delivery and toxicology a challenging task. The purpose of this chapter is to review the state-of-the-art advances on ONs for applications in inherited disorders and give an overview of what is known regarding their delivery and safety, based on preclinical and clinical studies.
Oligonucleotide therapy / Covello, Giuseppina; Denti, Michela Alessandra. - STAMPA. - (2017), pp. 1-27.
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| Titolo: | Oligonucleotide therapy |
| Autori: | Covello, Giuseppina; Denti, Michela Alessandra |
| Autori Unitn: | |
| Titolo del volume contenente il saggio: | Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders |
| Luogo di edizione: | Cham |
| Casa editrice: | Springer International Publishing AG |
| Anno di pubblicazione: | 2017 |
| ISBN: | 978-3-319-53455-8 |
| Handle: | http://hdl.handle.net/11572/175885 |
| Citazione: | Oligonucleotide therapy / Covello, Giuseppina; Denti, Michela Alessandra. - STAMPA. - (2017), pp. 1-27. |
| Appare nelle tipologie: | 02.1 Saggio su volume miscellaneo o Capitolo di libro (Essay or Book Chapter) |
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| File | Descrizione | Tipologia | Licenza | |
|---|---|---|---|---|
| Covello_book_2017_preprint.pdf | bozza con correzioni editoriali | Versione editoriale (Publisher’s layout) | Tutti i diritti riservati (All rights reserved) | Administrator |
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