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Related Concept Videos

Protein Dynamics in Living Cells01:19

Protein Dynamics in Living Cells

Different fluorescence-based techniques are used to study the protein dynamics in living cells. These techniques include FRAP, FRET, and PET.
Fluorescent recovery after photobleaching (FRAP) is a fluorescent-protein-based detection technique used to quantify protein movement rates within the cell. This method exposes a small portion of the cell to an intense laser beam. The laser beam causes permanent photobleaching of the fluorophore-tagged proteins in the exposed region. As the bleached...

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Related Experiment Video

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Real-time Imaging of Single Engineered RNA Transcripts in Living Cells Using Ratiometric Bimolecular Beacons
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Published on: August 6, 2014

Rapid Access to Photoswitchable RNA Binders: Fluorination Enhances Protein Rescue by Exon Inclusion.

Lei Zhang1, Nemanja Djokovic2, Alexander Herr3

  • 1Chemistry Department, Marburg University, Marburg, Germany.

Chemistry (Weinheim an Der Bergstrasse, Germany)
|July 12, 2026
PubMed
Summary

Researchers developed novel photoswitchable RNA binders for precise, light-controlled therapeutic intervention. This platform simplifies synthesis, enabling targeting of pre-messenger RNA (pre-mRNA) to restore survival motor neuron (SMN) protein levels.

Keywords:
RNA bindersSMAfluorinationphotoswitchessplicing

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Area of Science:

  • Medicinal Chemistry
  • Molecular Biology
  • RNA Therapeutics

Background:

  • Targeting RNA offers therapeutic potential but faces challenges due to RNA's structural diversity and transient functional motifs.
  • Developing selective small-molecule binders for RNA is complex, hindering rational drug design.
  • Existing photoswitchable RNA molecular tools are scarce and require difficult synthesis.

Purpose of the Study:

  • To develop a readily adaptable platform for synthesizing novel photoswitchable RNA binders.
  • To enable light-controlled regulation of RNA interactions for precise therapeutic intervention.
  • To target pre-messenger RNA (pre-mRNA) and restore functional survival motor neuron (SMN) protein levels.

Main Methods:

  • Development of a straightforward synthesis platform for photoswitchable RNA binders.
  • Design and evaluation of compounds incorporating fluorination and heteroaryl groups.
  • Utilizing computational studies and Nuclear Magnetic Resonance (NMR) spectroscopy for structure-activity relationship analysis.

Main Results:

  • Demonstrated successful synthesis of photoswitchable RNA binders with a readily adaptable platform.
  • Identified that fluorination and heteroaryl groups enhance binding affinity to double-stranded RNA (dsRNA).
  • Confirmed in-cellulo activity and rationalized molecular recognition and structure-activity relationships.

Conclusions:

  • The developed platform facilitates the straightforward synthesis of photoswitchable RNA binders.
  • These binders can target pre-mRNA, rescue exon inclusion, and restore SMN protein levels.
  • The findings provide a foundation for developing novel RNA-targeted therapeutics with light-controlled regulation.