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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 of RNA aptamer-based therapeutics.

Jiehua Zhou¹, Maggie L Bobbin, John C Burnett

¹Division of Molecular and Cellular Biology, Beckman Research Institute of City of Hope Duarte, CA, USA.

Frontiers in Genetics

|November 7, 2012

Summary

RNA aptamers offer dual therapeutic functions by blocking target proteins and delivering RNA interference (RNAi) payloads. These aptamer-siRNA chimeras show promise for treating cancers and neurological diseases.

Keywords:

RNA aptamers RNA interference small interfering RNA systematic evolution of ligands by exponential enrichment targeted delivery

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Area of Science:

Biochemistry and Molecular Biology
Biotechnology
Therapeutics Development

Background:

Aptamers are single-stranded nucleic acids with stable 3D structures, enabling specific target recognition and binding.
Nucleic acid aptamers have diverse applications in diagnostics, imaging, drug delivery, and therapeutics.
Their high specificity and affinity make aptamers effective in blocking or interrupting target protein functions.

Purpose of the Study:

To review the progress and therapeutic potential of RNA aptamers.
To highlight the use of cell-internalizing aptamers for targeted RNA interference (RNAi).
To discuss emerging aptamer-based therapeutics for cancer and neurological diseases.

Main Methods:

Review of current literature on aptamer technology and applications.
Analysis of aptamer-siRNA chimera design and function.
Discussion of clinical opportunities for aptamer-based therapies.

Main Results:

Aptamers can directly inhibit protein function, serving as therapeutic agents.
Aptamers targeting cell surface proteins can deliver therapeutics like small interfering RNAs (siRNA).
Aptamer-siRNA chimeras offer dual functions: receptor inhibition and intracellular mRNA targeting via RNAi.

Conclusions:

RNA aptamers represent a promising therapeutic modality with potential for treating various human diseases.
Cell-internalizing aptamers serve as effective delivery vehicles for targeted RNAi.
Emerging aptamer-based therapeutics present unique clinical opportunities, particularly for cancers and neurological disorders.