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Related Concept Videos

Riboswitches01:56

Riboswitches

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Riboswitches are non-coding mRNA domains that regulate the transcription and translation of downstream genes without the help of proteins. Riboswitches bind directly to a metabolite and can form unique stem-loop or hairpin structures in response to the amount of the metabolite present. They have two distinct regions – a metabolite-binding aptamer and an expression platform.
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In eukaryotic cells, nascent mRNA transcripts need to undergo many post-transcriptional modifications to reach the cell cytoplasm and translate into functional proteins. For a long time, transcription and pre-mRNA processing were considered two independent events that occur sequentially in the cell. However, it has now been well established that transcription and pre-mRNA processing are two simultaneous processes that are precisely regulated inside the cell.
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Regulation of Expression at Multiple Steps

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The gene expression in cells is regulated at different stages: (i) transcription, (ii) RNA processing, (iii) RNA localization, and (iv) translation. Transcriptional regulation is mediated by regulatory proteins such as transcription factors, activators, or repressors—these control gene expression by initiating or inhibiting the transcription of genes. Once a precursor or pre-mRNA is produced, it undergoes post-transcriptional modification, including 5' capping, splicing, and the...
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Alternative RNA splicing is the regulated splicing of exons and introns to produce different mature mRNAs from a single pre-mRNA. Unlike in constitutive splicing where a single gene produces a single type of mRNA, alternative splicing allows an organism to produce multiple proteins from a single gene and plays an important role in protein diversity.
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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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Optimization of Exon-Skipping Riboswitches and Their Applications to Control Mammalian Cell Fate.

Yoko Nomura1, Narae Kim1, Bochen Zhu1

  • 1Nucleic Acid Chemistry and Engineering Unit, Okinawa Institute of Science and Technology Graduate University Onna, Okinawa 9040495, Japan.

ACS Synthetic Biology
|September 25, 2024
PubMed
Summary

Researchers optimized mammalian riboswitches for gene expression control. These RNA-based systems offer precise chemical regulation of cell phenotype without protein factors, enhancing medical and biotech applications.

Keywords:
alternative splicingaptamercell-fate controlkill switchriboswitch

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Area of Science:

  • Synthetic Biology
  • Molecular Biology
  • Biotechnology

Background:

  • Mammalian riboswitches offer protein-free regulation of transgene expression, useful in medicine and biotechnology.
  • Current limitations include lack of suitable small molecules, aptamers, and modest performance.
  • This study addresses these limitations by optimizing riboswitch design for enhanced function.

Purpose of the Study:

  • To systematically optimize a mammalian riboswitch for regulating gene expression via exon skipping.
  • To fine-tune riboswitch characteristics by modulating aptamer base stem stability.
  • To demonstrate the application of the optimized riboswitch in controlling mammalian cell differentiation and apoptosis.

Main Methods:

  • Designed and optimized a riboswitch controlling exon skipping through an RNA aptamer binding ASP2905.
  • Investigated two strategies to modulate aptamer base stem stability for fine-tuning riboswitch performance.
  • Generated cell lines (mouse embryonic stem cells, human embryonic kidney cells) for demonstrating riboswitch function.

Main Results:

  • Successfully optimized a mammalian riboswitch design for improved performance.
  • Demonstrated tight chemical control over transgene expression (Myod1, BAX) in mammalian cells.
  • Achieved chemically induced differentiation of stem cells and apoptosis in kidney cells.

Conclusions:

  • Optimized mammalian riboswitches provide precise, protein-free chemical control over gene expression and cell phenotype.
  • This advancement expands the potential of RNA-based regulatory systems in biotechnology and medicine.
  • The developed riboswitches offer a versatile tool for applications requiring inducible gene expression.