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siRNA - Small Interfering RNAs

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...

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Exploring Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins

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Aptamer-siRNA Chimeras: Discovery, Progress, and Future Prospects.

Sven Kruspe¹, Paloma H Giangrande^2,3

¹Internal Medicine, University of Iowa, Iowa City, IA 52242, USA. sven-kruspe@uiowa.edu.

|August 10, 2017

Summary

Aptamers are synthetic nucleic acid ligands that effectively deliver therapeutic oligonucleotides like small interfering RNAs (siRNAs). Recent advances enable cell-specific aptamer selection for targeted drug delivery in cancer and antiviral therapies.

Keywords:

RNA interference aptamers targeted therapy

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Development of Cell-type specific anti-HIV gp120 aptamers for siRNA delivery

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Development of Cell-type specific anti-HIV gp120 aptamers for siRNA delivery

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Published on: June 23, 2011

Area of Science:

Biotechnology and Molecular Medicine
Nucleic Acid Therapeutics
Drug Delivery Systems

Background:

Synthetic nucleic acid ligands, known as aptamers, are increasingly utilized as delivery vehicles for oligonucleotide-based therapeutics.
Aptamers offer a promising platform for targeted delivery of drugs like small interfering RNAs (siRNAs) to specific cells.

Purpose of the Study:

To review advancements in aptamer selection technologies for identifying cell-specific, internalizing aptamers.
To summarize progress in aptamer-siRNA conjugate designs for therapeutic applications.
To highlight challenges and future prospects for aptamer-targeted siRNA drugs in clinical development.

Main Methods:

Review of recent literature on aptamer selection methodologies.
Analysis of proof-of-concept studies demonstrating aptamer-mediated siRNA delivery.
Discussion of current aptamer-siRNA conjugate designs and their applications.

Main Results:

Progress in aptamer selection has yielded cell-specific aptamers capable of internalization.
Aptamer-siRNA conjugates show potential in diverse therapeutic areas, including cancer and viral infections.
Established proof-of-concept studies validate aptamer-based siRNA delivery systems.

Conclusions:

Aptamers are effective tools for targeted delivery of therapeutic oligonucleotides, particularly siRNAs.
Continued development in aptamer selection and conjugate design enhances their therapeutic potential.
Further research and clinical development are necessary to realize the full promise of aptamer-targeted siRNA drugs.