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Coupling Aptamers to Short Interfering RNAs as Therapeutics
Laura Cerchia1, Carla Lucia Esposito2, Simona Camorani2,3
1Istituto per l'Endocrinologia e l'Oncologia Sperimentale del CNR "G. Salvatore", Via S. Pansini 5, 80131 Naples, Italy. cerchia@unina.it.
Pharmaceuticals (Basel, Switzerland)
|October 11, 2016
Summary
RNA therapeutics offer promising treatments but face delivery challenges. Chemical modifications and aptamer-based bioconjugates enhance RNA stability and cell targeting for gene expression therapies.
Area of Science:
- Biotechnology
- Molecular Biology
- Therapeutics Development
Background:
- RNA-based therapeutics, including miRNAs, siRNAs, and aptamers, show great potential.
- Significant barriers hinder RNA therapeutic efficacy, such as nuclease degradation and poor cellular uptake.
- Targeted delivery and intracellular access remain critical challenges for RNA-based drugs.
Approach:
- Chemical modifications stabilize small RNAs, improving their integrity and functional activity.
- RNA aptamers are engineered for precise recognition of cell surface targets.
- Molecular bioconjugates are developed to facilitate targeted delivery and cellular internalization of RNA therapeutics.
Key Points:
- Chemical modifications enhance the stability and conformation of small RNAs.
- Aptamers provide specific cell surface targeting capabilities.
- Bioconjugate strategies enable targeted delivery and internalization of RNA molecules.
Conclusions:
- Recent advances in RNA modification and bioconjugation address key therapeutic delivery barriers.
- Targeted delivery systems improve the potential of RNA therapeutics for gene expression modulation.
- Molecular bioconjugates represent a promising strategy for advancing RNA-based therapies.
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