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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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Switching behavior in Bipolar Junction Transistors (BJTs) is a fundamental aspect utilized in various electronic circuits, particularly for digital logic applications like switches and amplifiers. In a typical switching circuit, a BJT alternates between cut-off and saturation modes, corresponding to the "off" and "on" states, respectively, thus behaving like an ideal switch.
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Anti-gene oligonucleotides targeting Friedreich's ataxia expanded GAAâ‹…TTC repeats increase Frataxin expression.

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Related Experiment Video

Updated: Jan 20, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Antisense Oligonucleotides for Splice Modulation: Assessing Splice Switching Efficacy.

Cristina S J Rocha1

  • 1Clinical Pharmacology & Safety Sciences, BioPharmaceuticals R&D, AstraZeneca, Gothenburg, Sweden. cristina.rocha@astrazeneca.com.

Methods in Molecular Biology (Clifton, N.J.)
|August 15, 2019
PubMed
Summary
This summary is machine-generated.

This study presents an accessible protocol for evaluating splice switching oligonucleotide efficacy. Gene expression analysis is used to assess drug potency and efficacy in molecular biology labs.

Keywords:
Agarose electrophoresisAntisense oligonucleotidesRNA isolationRT-PCRSplice modulation efficacySplice switching oligonucleotides

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Area of Science:

  • Molecular Biology
  • Pharmacology
  • Genetics

Background:

  • Oligonucleotide therapies are increasingly approved, offering a distinct mechanism of action by targeting RNA.
  • Unlike traditional small molecules acting on proteins, these therapies modulate gene expression.
  • Evaluating drug potency, encompassing target engagement and efficacy, is crucial for therapeutic development.

Purpose of the Study:

  • To provide a straightforward protocol for assessing splice switching oligonucleotide (SSO) efficacy.
  • To enable evaluation of SSO potency and efficacy using accessible gene expression analysis.
  • To facilitate SSO efficacy assessment in standard molecular biology laboratories.

Main Methods:

  • The protocol details steps from sample preparation to data analysis for SSO efficacy evaluation.
  • It focuses on utilizing gene expression analysis for assessing both affinity and efficacy.
  • The method is designed for ease of application without requiring advanced laboratory equipment.

Main Results:

  • The described protocol allows for the evaluation of splice switching oligonucleotide efficacy.
  • Gene expression analysis serves as a reliable method to assess SSO affinity and efficacy.
  • The protocol is validated for its applicability in standard molecular biology settings.

Conclusions:

  • This chapter offers a practical and accessible method for determining splice switching oligonucleotide efficacy.
  • The protocol empowers researchers to evaluate SSO performance using readily available techniques.
  • Accessible evaluation of oligonucleotide therapy efficacy is vital for advancing this therapeutic modality.