Noncoding RNA therapeutics - challenges and potential solutions.
Nat Rev Drug Discov
; 20(8): 629-651, 2021 08.
Article
in English
| MEDLINE | ID: covidwho-1341001
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.
Full text:
Available
Collection:
International databases
Database:
MEDLINE
Main subject:
Genetic Therapy
/
Molecular Targeted Therapy
/
RNA, Long Noncoding
/
Neoplasms
Type of study:
Prognostic study
/
Randomized controlled trials
Limits:
Animals
/
Humans
Language:
English
Journal:
Nat Rev Drug Discov
Journal subject:
Pharmacology
/
Drug Therapy
Year:
2021
Document Type:
Article
Affiliation country:
S41573-021-00219-z
Similar
MEDLINE
...
LILACS
LIS