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Related Concept Videos

RNA Splicing01:32

RNA Splicing

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Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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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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Alternative RNA Splicing02:18

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RNA Interference01:23

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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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Related Experiment Video

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RNA Interference in Ticks
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RNA Trans-Splicing Modulation via Antisense Molecule Interference.

Bernadette Liemberger1, Josefina Piñón Hofbauer2, Verena Wally3

  • 1EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital of the Paracelsus Medical University, 5020 Salzburg, Austria. b.liemberger@salk.at.

International Journal of Molecular Sciences
|March 10, 2018
PubMed
Summary
This summary is machine-generated.

Researchers developed a new screening system to improve RNA trans-splicing efficiency for genetic diseases. This system identified antisense molecules that enhance RNA editing, potentially aiding clinical applications for inherited disorders.

Keywords:
KRT14RNA therapyRNA trans-splicingantisense moleculesepidermolysis bullosafluorescence-based screening system

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Identification of Alternative Splicing and Polyadenylation in RNA-seq Data
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Identification of Alternative Splicing and Polyadenylation in RNA-seq Data

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Area of Science:

  • Molecular Biology
  • Genetic Engineering
  • RNA Therapeutics

Background:

  • RNA trans-splicing is a promising gene editing tool for correcting mutations at the pre-mRNA level.
  • Current RNA trans-splicing efficiency requires improvement for widespread clinical use in genetic diseases.

Purpose of the Study:

  • To develop a fluorescence-based screening system for enhancing RNA trans-splicing efficiency.
  • To identify antisense molecules that improve the efficiency of a KRT14-specific RNA trans-splicing molecule.

Main Methods:

  • A modified, fluorescence-based screening system was established for generating and analyzing antisense molecules.
  • The system was used to screen for molecules that enhance RNA reprogramming efficiency.
  • Identified molecules were analyzed for their ability to increase trans-splicing efficiency.

Main Results:

  • The screening system successfully identified several antisense RNAs and short oligonucleotides.
  • These molecules demonstrated an ability to significantly increase RNA trans-splicing efficiency.
  • The identified molecules act as splicing modulators, enhancing the therapeutic potential of RNA trans-splicing.

Conclusions:

  • Short antisense molecules can function as splicing modulators to boost RNA trans-splicing efficiency.
  • This approach may achieve therapeutic levels of RNA editing for genetic predispositions, especially dominant inherited diseases.
  • The developed screening system facilitates the discovery of novel RNA editing enhancers.