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Designer antisense-circular RNAs (AS circRNAs)for the inhibition of pre-mRNA splicing and mRNA translation


Abstract

We present two new lines of designer antisense-circRNAs (AS-circRNAs), which are capable of influencing pre-mRNA splicing and mRNA translation.


Background

Antisense approaches are an innovative and growing field of gene therapies. Best known are therapies based on linear antisense oligonucleotides (ASOs), but they show deficits in efficiency, stability, and off-target effects.


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Solution

Circular RNAs (circRNAs), which are generated from pre-mRNAs by an alternative splicing mechanism called back-splicing, show higher stability than their linear counterparts. This and other emerging features like miRNA sponging, protein sponging, and protein translation, make them attractive for novel RNA therapeutics.


Advantages

CircRNAs show following advantages:

  • much more stable than corresponding linear RNA
  • high binding specificity
  • highly flexible in sequence design and target specificity

Scope of application

  • RNA therapeutics
  • Cancer treatment/therapy
  • Pharmaceutical compositions
  • Molecular applications

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Anouschka Ulherr
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anouschka.ulherr@transmit.de

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Development status

Feasibility


Keywords

molecular medicine, antisense circRNAs, genetic disease, cancer, RNA therapeu-tics

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