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Noncoding RNA therapeutics - challenges and potential solutions.

ABSTRACT: Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of cancers, as well as many other diseases. Over the past decade, substantial effort has been made towards the clinical application of RNA-based therapeutics, employing mostly antisense oligonucleotides and small interfering RNAs, with several gaining FDA approval. However, trial results have so far been ambivalent, with some studies reporting potent effects whereas others demonstrated limited efficacy or toxicity. Alternative entities such as antimiRNAs are undergoing clinical testing, and lncRNA-based therapeutics are gaining interest. In this Perspective, we discuss key challenges facing ncRNA therapeutics - including issues associated with specificity, delivery and tolerability - and focus on promising emerging approaches that aim to boost their success.

SUBMITTER: Winkle M

PROVIDER: S-EPMC8212082 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature

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Noncoding RNA therapeutics - challenges and potential solutions.

Winkle Melanie M El-Daly Sherien M SM Fabbri Muller M Calin George A GA

Nature reviews. Drug discovery 20210618 8

Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of cancers, as well as many other diseases. Over the past decade, substantial effort has been made towards the clinical application of RNA-based therapeutics, employing mostly antisense oligonucleotides and small interfering RNAs, with several gaining FDA approval. However, trial results have so far been ambivalent, with some studies ...[more]

PMID: 34145432

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Dataset Information

Noncoding RNA therapeutics - challenges and potential solutions.

Publications

Noncoding RNA therapeutics - challenges and potential solutions.

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