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People tend to know what behavior is expected of them in specific, familiar settings. A script is a person’s knowledge about the sequence of events expected in a specific setting (Schank & Abelson, 1977). Essentially, scripts are a particular kind of schema, one containing default values for the features within an event. In the restaurant example, the script's features include the props (e.g., tables, menu, food, and money), the roles to be played (e.g., customer and waiter),...
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Related Experiment Video

Updated: Jan 27, 2026

In Vivo Intracellular Recording of Type-Identified Rat Spinal Motoneurons During Trans-Spinal Direct Current Stimulation
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Rewriting the (tran)script: Application to spinal muscular atrophy.

Hasane Ratni1, Lutz Mueller1, Martin Ebeling1

  • 1F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development, Roche Innovation Center Basel, Basel, Switzerland.

Progress in Medicinal Chemistry
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PubMed
Summary
This summary is machine-generated.

Targeting RNA offers new therapeutic strategies for diseases. Splicing modulation, using splice switching oligonucleotides and small molecules, has shown success in treating spinal muscular atrophy (SMA).

Keywords:
Alternative splicingRNASafetySelectivitySmall moleculesSpinal muscular atrophySplice switching oligonucleotides

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Area of Science:

  • RNA therapeutics
  • Gene expression modulation

Background:

  • Targeting RNA expands therapeutic possibilities for various diseases.
  • Pre-mRNA splicing modulation is a viable strategy to control cellular processes by altering exon inclusion/exclusion.
  • Recent breakthroughs in spinal muscular atrophy (SMA) treatment demonstrate the potential of this approach.

Purpose of the Study:

  • To review the concept and progress of splicing modulation as a therapeutic strategy.
  • To highlight recent advancements in treating spinal muscular atrophy (SMA) using novel modalities.
  • To discuss the critical requirements for selectivity and safety in splicing-targeting drugs.

Main Methods:

  • Review of scientific literature on RNA targeting and splicing modulation.
  • Highlighting therapeutic modalities like splice switching oligonucleotides and small molecules.
  • Discussion of selectivity and safety aspects of drug development.

Main Results:

  • Significant progress has been made in targeting RNA for therapeutic purposes.
  • Successful application of splice switching oligonucleotides and small molecules in treating spinal muscular atrophy (SMA).
  • Demonstration of the feasibility of achieving selectivity and addressing safety concerns.

Conclusions:

  • Splicing modulation represents a powerful therapeutic strategy with demonstrated success in SMA.
  • The development of targeted RNA therapies is rapidly advancing.
  • Transformational medicines for SMA are likely precursors to broader applications of RNA-targeting drugs.