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Overexpressing Long Noncoding RNAs Using Gene-activating CRISPR
Published on: March 1, 2019
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The potential of long noncoding RNA therapies
Tim R Mercer1, Trent Munro1, John S Mattick2
1Australian Institute for Biotechnology and Nanomaterials, University of Queensland, St Lucia, Australia.
Trends in Pharmacological Sciences
|February 14, 2022
Summary
Natural long noncoding RNAs (lncRNA) offer a blueprint for designing novel RNA therapies. Advances in RNA technology pave the way for exploiting lncRNA structures in synthetic biology and medicine.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- The human genome encodes numerous long noncoding RNAs (lncRNAs) involved in critical cellular processes.
- lncRNAs exert functions through sequence motifs and secondary structures, influencing gene regulation.
- These molecules play roles in cell biology, development, and disease pathogenesis.
Purpose of the Study:
- To propose the exploration and utilization of natural lncRNA structures for designing synthetic RNA therapies.
- To leverage insights from existing RNA-based therapies to predict the properties of future lncRNA therapeutics.
Main Methods:
- Analysis of lncRNA sequence motifs and secondary structures.
- Review of advances in RNA stability, immunogenicity, manufacture, and delivery.
- Application of synthetic biology principles to RNA device engineering.
Main Results:
- Natural lncRNA structures can be rationally designed into therapeutic RNA devices.
- Existing RNA therapy advancements provide a framework for anticipating lncRNA therapy pharmacology.
- Potential for lncRNA therapies to address unmet clinical needs.
Conclusions:
- Natural lncRNA structures represent a promising resource for developing innovative RNA-based therapeutics.
- The field of RNA therapeutics is rapidly advancing, enabling the translation of lncRNA research into clinical applications.
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