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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 Splicing02:18

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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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Chromatin Structure Regulates pre-mRNA Processing02:41

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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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A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is comprised  of nucleotides and proteins are comprised of amino acids, a mediator is required to convert the information encoded in DNA into proteins. This mediator is the messenger RNA (mRNA). mRNA copies the blueprint from DNA by a process called transcription. In eukaryotes, transcription occurs in the nucleus by complementary base-pairing with the DNA template. The mRNA is then...
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Pre-mRNA Processing: RNA Splicing01:36

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A Reporter Based Cellular Assay for Monitoring Splicing Efficiency
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[RNA splicing modulation: Therapeutic progress and perspectives].

Amel Saoudi1, Aurélie Goyenvalle2

  • 1Université Paris-Saclay, UVSQ, Inserm, END-ICAP, Handicap neuromusculaire - physiopathologie, biothérapie et pharmacologie appliquées, 78000 Versailles, France - Université Paris-Saclay, CNRS, Institut des neurosciences Paris Saclay, 91190 Gif-sur-Yvette, France.

Medecine Sciences : M/S
|June 28, 2021
PubMed
Summary
This summary is machine-generated.

RNA modulation therapies offer new hope for hereditary diseases caused by aberrant splicing. Antisense oligonucleotides (ASOs) show promise, but delivery challenges drive research into new chemistries and delivery systems.

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Area of Science:

  • Genetics and Genomics
  • Molecular Biology
  • Therapeutics

Background:

  • Hereditary diseases are increasingly linked to aberrant RNA splicing.
  • RNA modulation therapies, particularly antisense oligonucleotides (ASOs), are emerging as promising treatments.
  • Approved ASO therapies exist for Duchenne muscular dystrophy and spinal muscular atrophy.

Purpose of the Study:

  • To review recent advances in splicing therapies for neuromuscular diseases.
  • To discuss the benefits and limitations of current RNA modulation strategies.
  • To highlight ongoing research in improving ASO delivery and efficacy.

Main Methods:

  • Review of current literature on splicing therapies.
  • Focus on antisense oligonucleotide (ASO) technology.
  • Discussion of alternative delivery systems and small molecules.

Main Results:

  • Several ASO-based drugs have been approved for genetic neuromuscular disorders.
  • Significant progress has been made in chemical modifications and design of ASOs.
  • Limited biodistribution remains a key challenge for systemic ASO administration.

Conclusions:

  • Splicing-based therapies, especially ASOs, represent a significant advancement in treating hereditary diseases.
  • Overcoming delivery challenges is crucial for broader clinical application.
  • Continued research into novel chemistries and delivery methods is essential for optimizing these therapies.