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Non-coding RNAs: Therapeutic Strategies and Delivery Systems
1Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA. hling@mdanderson.org.
Advances in Experimental Medicine and Biology
|August 31, 2016
Summary
Noncoding RNAs, including microRNAs and long noncoding RNAs, are promising therapeutic targets for cancer. Effective delivery systems are crucial for developing novel noncoding RNA therapeutics.
Area of Science:
- Genetics
- Molecular Biology
- Pharmacology
Background:
- The human genome extensively transcribes noncoding RNAs, such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs).
- Dysregulation of miRNAs in cancers has spurred interest in their therapeutic potential as drugs and drug targets.
- lncRNAs are emerging as therapeutic targets due to their roles in complex phenotypes and protein output regulation.
Purpose of the Study:
- To discuss general therapeutic strategies targeting noncoding RNAs.
- To review delivery systems for enhancing noncoding RNA therapeutic efficacy.
- To offer perspectives on noncoding RNA-targeting agents for colorectal cancer.
Main Methods:
- Review of preclinical studies on oligonucleotide-based miRNA mimicking and blocking.
- Analysis of chemical modifications and delivery systems for nucleic acid therapeutics.
- Exploration of therapeutic strategies for noncoding RNAs.
Main Results:
- miRNA therapeutics have entered clinical trials for various diseases, including cancer.
- Challenges remain in the effective and safe delivery of nucleic acid therapeutics.
- Various delivery systems and chemical modifications are being developed to improve efficacy and reduce side effects.
Conclusions:
- Noncoding RNAs represent a significant area for therapeutic development.
- Advancements in delivery systems are critical for realizing the potential of noncoding RNA therapeutics.
- Future development of noncoding RNA targeting agents holds promise for diseases like colorectal cancer.
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