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

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

Published on: August 9, 2019

Current progress on aptamer-targeted oligonucleotide therapeutics.

Justin P Dassie¹, Paloma H Giangrande

¹Department of Internal Medicine, University of Iowa, 375 Newton Rd, 5202 MERF, Iowa City, IA 52242, USA.

Therapeutic Delivery

|December 6, 2013

Summary

Aptamers offer a promising solution for delivering therapeutic RNA interference (RNAi) drugs like small interfering RNAs (siRNAs) to target cells, overcoming major delivery challenges for treating diseases.

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Area of Science:

Biotechnology
Molecular Biology
Drug Delivery

Background:

The RNA interference (RNAi) pathway holds therapeutic potential for various diseases.
Effective delivery of nucleic acid drugs, such as small interfering RNAs (siRNAs), remains a significant obstacle for clinical application.

Purpose of the Study:

To review recent advancements in using cell-internalizing aptamers for delivering therapeutic oligonucleotides.
To discuss aptamer-siRNA chimera technology and aptamer-functionalized nanoparticles for siRNA delivery.
To highlight challenges and future prospects for clinical translation of aptamer-targeted oligonucleotide drugs.

Main Methods:

Review of recent literature on aptamer-based delivery systems.
Discussion of aptamer-siRNA chimeras and aptamer-functionalized nanoparticles.
Analysis of challenges and future directions in clinical translation.

Main Results:

Cell-internalizing aptamers are effective delivery vehicles for therapeutic oligonucleotides, including siRNAs, miRNAs, anti-miRs, and antisense oligos.
Aptamer-siRNA chimera technology and aptamer-functionalized nanoparticles show promise for enhanced siRNA delivery.
Significant progress has been made in developing aptamer-targeted oligonucleotide drug delivery systems.

Conclusions:

Aptamers represent a viable strategy to overcome the delivery barriers of oligonucleotide therapeutics.
Further research and development are needed to address challenges for successful clinical translation.
Aptamer-based delivery systems offer a promising future for treating a wide range of human diseases.