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Exploring Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins
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Aptamers as Therapeutics.

Shahid M Nimjee1, Rebekah R White2, Richard C Becker3

  • 1Department of Neurological Surgery, The Ohio State University Wexner Medical Center, Columbus, Ohio 43210;

Annual Review of Pharmacology and Toxicology
|January 8, 2017
PubMed
Summary
This summary is machine-generated.

Aptamers, or nucleic acid drugs, are developed using SELEX. These therapies are optimized for clinical use and show promise for treating diseases like cancer and visual disorders.

Keywords:
SELEXantidoteaptamerpharmacologytherapeutics

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Pharmacology

Background:

  • Aptamers are single-stranded nucleic acid therapies that bind and inhibit protein targets.
  • Systematic Evolution of Ligands by Exponential Enrichment (SELEX) is a common method for aptamer production.
  • Therapeutic aptamers have a 25-year history, with one approved drug and many in development.

Purpose of the Study:

  • To provide a historical overview of aptamers and SELEX.
  • To describe the pharmacological development and optimization of aptamer therapeutics.
  • To review the potential applications of aptamers in treating various diseases.

Main Methods:

  • Review of historical data and scientific literature on aptamers and SELEX.
  • Analysis of aptamer modification strategies for therapeutic enhancement.
  • Compilation of research on aptamer applications in disease treatment.

Main Results:

  • Aptamers offer tailored therapeutic properties through pharmacological revision.
  • Significant progress has been made in aptamer development, moving from discovery to clinical application.
  • Aptamers demonstrate potential in treating conditions such as visual disorders, thrombosis, and cancer.

Conclusions:

  • Aptamers represent a significant class of therapeutic molecules with a growing clinical presence.
  • The optimization of aptamers through SELEX and pharmacological revision is key to their therapeutic success.
  • Further research and development are expected to expand the clinical utility of aptamers across diverse diseases.