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siRNA - Small Interfering RNAs02:30

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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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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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Updated: Mar 6, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Delivery is key: lessons learnt from developing splice-switching antisense therapies.

Caroline Godfrey1, Lourdes R Desviat2, Bård Smedsrød3

  • 1Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.

EMBO Molecular Medicine
|March 15, 2017
PubMed
Summary
This summary is machine-generated.

Splice-switching antisense therapy shows promise but faces challenges in delivering antisense oligonucleotides to target tissues. This review discusses current methods and proposes recommendations to advance therapeutic applications.

Keywords:
RNA therapyantisense oligonucleotidesdeliverypre‐clinical modelstoxicity

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Therapeutics

Background:

  • Splice-switching antisense therapy holds significant therapeutic potential, supported by extensive pre-clinical data and ongoing clinical trials.
  • The clinical translation of this promising approach is hindered by the inefficient delivery of antisense oligonucleotides (ASOs) to target tissues following systemic administration.

Purpose of the Study:

  • To critically evaluate standard methodologies in pre-clinical development of antisense oligonucleotide therapies.
  • To identify knowledge gaps and key challenges impeding the clinical application of splice-switching antisense therapy.
  • To provide recommendations for future research to enhance the efficacy and application of therapeutic antisense oligonucleotides.

Main Methods:

  • Discussion and critical review of existing pre-clinical development methodologies for antisense oligonucleotide therapies.
  • Analysis of current knowledge gaps and challenges in the field of splice-switching antisense therapy.
  • Formulation of recommendations based on expert consensus.

Main Results:

  • Standard methodologies for pre-clinical development require re-evaluation for their validity and relevance.
  • Significant knowledge gaps exist concerning optimal delivery strategies and target engagement.
  • Key challenges include achieving sufficient tissue-specific delivery and mitigating off-target effects.

Conclusions:

  • Addressing the challenge of antisense oligonucleotide delivery is crucial for realizing the therapeutic potential of splice-switching antisense therapy.
  • Future research should focus on innovative delivery systems and robust validation methods.
  • Implementing the proposed recommendations can accelerate the clinical application of these advanced therapeutics.