Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

RNA Splicing01:32

RNA Splicing

57.2K
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...
57.2K
RNA Interference01:23

RNA Interference

26.5K
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.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
26.5K
RNA Editing02:23

RNA Editing

9.2K
RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
9.2K
Experimental RNAi02:15

Experimental RNAi

6.3K
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...
6.3K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

De novo design of RNA pseudoknots with deep learning.

bioRxiv : the preprint server for biology·2026
Same author

A novel splice site variant in DEGS1 leads to aberrant splicing and loss of DEGS1 enzyme activity, a VUS resolved.

Human genetics·2026
Same author

Structures of nucleotide-bound human telomerase at several steps of its telomeric DNA repeat addition cycle.

Nature communications·2026
Same author

Template-based RNA structure prediction advanced through a blind code competition.

bioRxiv : the preprint server for biology·2026
Same author

Therapeutic 6-thio-deoxyguanosine inhibits telomere elongation in cancer cells by inducing a non-productive stalled telomerase complex.

Nature communications·2025
Same author

IGF2BP3 remodels the microRNA targeting landscape in MLL-AF4 leukemia.

bioRxiv : the preprint server for biology·2025
Same journal

Optimized tRNA structure-seq reveals robust tRNA secondary structures in <i>S. cerevisiae</i> under mild stress conditions.

RNA (New York, N.Y.)·2026
Same journal

SERIPH: A Two-Step Extraction Protocol for Selective Enrichment of Semi-Extractable RNAs.

RNA (New York, N.Y.)·2026
Same journal

Reduced Sensitivity to RNA Structural Differences Distinguishes Eukaryotic Pus4 from Bacterial TruB.

RNA (New York, N.Y.)·2026
Same journal

Puf3 contributes to changes in mRNA solubility, translation elongation dynamics at rare arginine codons and loss of protein homeostasis in cells lacking Not4.

RNA (New York, N.Y.)·2026
Same journal

RBM38 Regulates HORMAD1 Splicing to Enhances MEK Inhibitor Sensitivity in Breast Cancer.

RNA (New York, N.Y.)·2026
Same journal

EF-P Inhibits Ribosomal α-Hydroxy Acid Incorporation: Strategic tRNA Body Selection for Co-incorporating α-Hydroxy Acids and Nonproteinogenic Amino Acids into Depsipeptides.

RNA (New York, N.Y.)·2026
See all related articles

Related Experiment Video

Updated: Sep 20, 2025

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
10:06

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells

Published on: April 26, 2017

9.1K

OpenASO: RNA Rescue-designing splice-modulating antisense oligonucleotides through community science.

Victor Tse1,2, Martin Guiterrez1,2, Jill Townley3

  • 1Department of Molecular, Cell and Developmental Biology, University of California Santa Cruz, Santa Cruz, California 95064, USA.

RNA (New York, N.Y.)
|May 27, 2025
PubMed
Summary
This summary is machine-generated.

Citizen scientists can accelerate the discovery of splice-modulating antisense oligonucleotides (ASOs) for treating genetic diseases. Crowdsourced ASOs show significant potential in enhancing gene splicing for conditions like hemophilia A.

Keywords:
RNA splicingRNA structureantisense oligonucleotidescommunity sciencedrug discoveryhemophilia A

More Related Videos

An Oligonucleotide-based Tandem RNA Isolation Procedure to Recover Eukaryotic mRNA-Protein Complexes
09:45

An Oligonucleotide-based Tandem RNA Isolation Procedure to Recover Eukaryotic mRNA-Protein Complexes

Published on: August 18, 2018

11.2K
A Reporter Based Cellular Assay for Monitoring Splicing Efficiency
08:53

A Reporter Based Cellular Assay for Monitoring Splicing Efficiency

Published on: September 15, 2021

2.9K

Related Experiment Videos

Last Updated: Sep 20, 2025

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
10:06

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells

Published on: April 26, 2017

9.1K
An Oligonucleotide-based Tandem RNA Isolation Procedure to Recover Eukaryotic mRNA-Protein Complexes
09:45

An Oligonucleotide-based Tandem RNA Isolation Procedure to Recover Eukaryotic mRNA-Protein Complexes

Published on: August 18, 2018

11.2K
A Reporter Based Cellular Assay for Monitoring Splicing Efficiency
08:53

A Reporter Based Cellular Assay for Monitoring Splicing Efficiency

Published on: September 15, 2021

2.9K

Area of Science:

  • RNA Therapeutics
  • Genetics
  • Bioinformatics

Background:

  • Splice-modulating antisense oligonucleotides (ASOs) are emerging RNA-based therapeutics for human diseases.
  • Traditional methods for discovering splice-modulating ASOs are resource-intensive and time-consuming.
  • Previous work identified ASOs targeting intronic RNA structures to correct pathogenic variants of F8 exon 16 in hemophilia A.

Purpose of the Study:

  • To develop a novel, efficient approach for discovering splice-modulating ASOs by integrating RNA structure prediction and citizen science.
  • To evaluate the efficacy of ASOs designed through a crowdsourcing platform in enhancing the splicing of a deficient F8 exon 16 variant.

Main Methods:

  • Utilized data-driven RNA structure prediction algorithms.
  • Leveraged a community science platform (Eterna OpenASO challenge) for ASO design.
  • Assessed the impact of designed ASOs on F8 exon 16 splicing in a cellular model.

Main Results:

  • 25% of top-ranked ASOs designed by citizen scientists significantly improved exon 16 splicing.
  • Combinations of ASOs designed by Eterna players demonstrated additive effects in enhancing splicing efficiency.
  • The crowdsourced approach successfully identified functional splice-modulating ASOs.

Conclusions:

  • Crowdsourcing ASO design through citizen science offers a promising strategy to accelerate the discovery of novel RNA-based therapeutics.
  • This approach can complement traditional methods, potentially reducing costs and time in drug development for genetic disorders.
  • The findings support the use of community science in advancing precision medicine and treating human diseases like hemophilia A.