ArticleNature2024
Split intein-mediated protein trans-splicing to express large dystrophins.
Abstract read
In one paragraph
Article in Nature, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.
0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed
–field-weighted citation impact
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
40 citing papers in PubMed.
- Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2026Article
- A protein chaperone can stabilize the intein-containing precursor to indirectly promote protein splicing.Protein science : a publication of the Protein Society · 2026Article
- Duchenne muscular dystrophy: from gene to gene-ius therapies.Skeletal muscle · 2026Review
- Organoids - the future of pre-clinical development of AAV gene therapy for CNS disorders.Gene therapy · 2026Review
- Traceless protein semisynthesis in cells using the promiscuous ultra-fast split intein NrdJ-1.Chemical science · 2026Article
- The atomic structure of human dystrophin spectrin-like repeat 24.Acta crystallographica. Section F, Structural biology communications · 2026Article
- CHARIOT-AAV: Conjugation of diverse vectors to adeno-associated viruses for delivery of large genes.bioRxiv : the preprint server for biology · 2026Article
- Design and initial characterization of a novel mini-promoter for gene therapies targeting the central nervous system.Molecular therapy. Advances · 2026Article
- Traceless protein semi-synthesis in cells using the promiscuous ultra-fast split intein NrdJ-1.bioRxiv : the preprint server for biology · 2026Article
- Molecular Mechanisms and Therapeutic Strategies in Heart Failure Due to Dystrophin Deficiency: A Comprehensive Review.Reviews in cardiovascular medicine · 2026Review
- From oversight to insight: the curious case of the endothelial insulin receptor.Blood vessels, thrombosis & hemostasis · 2026Review
- Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling.Journal of cachexia, sarcopenia and muscle · 2026Review
- Proteomics-based evaluation of AAV dystrophin gene therapy outcomes in mdx skeletal muscle.JCI insight · 2026Article
- Coiled-coil heterodimer-mediated split base editing systems enable flexible and robust nucleotide substitutions.Nature communications · 2026Article
- Protein engineering: status report.Protein engineering, design & selection : PEDS · 2026Review
- From molecular correction to functional rescue: delivery, tissue state, and immunobiology as the principal constraints on dystrophin-restoring therapy in Duchenne muscular dystrophy.Frontiers in pharmacology · 2026Review
- Efficient kidney gene transfer and proximal tubule transduction using self-complementary AAV.cc47 vectors.Molecular therapy. Methods & clinical development · 2025Article
- Genetic strategies for therapy of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids · 2025Review
- Lysosomal damage is a therapeutic target in Duchenne muscular dystrophy.Science advances · 2025Article
- Cell therapy for Duchenne muscular dystrophy: promises, challenges, and controversies.Cellular and molecular life sciences : CMLS · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Hichem TasfaoutDepartment of Neurology, University of Washington School of Medicine, Seattle, WA, USA. tasfaout@uw.edu.ORCID 0000-0003-1902-606X
Christine L HalbertDepartment of Neurology, University of Washington School of Medicine, Seattle, WA, USA.
Timothy S McMillenDepartment of Bioengineering, College of Engineering and School of Medicine, University of Washington, Seattle, WA, USA.
James M AllenDepartment of Neurology, University of Washington School of Medicine, Seattle, WA, USA.
Theodore R ReyesDepartment of Neurology, University of Washington School of Medicine, Seattle, WA, USA.
Galina V FlintDepartment of Bioengineering, College of Engineering and School of Medicine, University of Washington, Seattle, WA, USA.
Dirk GrimmDepartment of Infectious Diseases/Virology, Section Viral Vector Technologies, Medical Faculty and Faculty of Engineering Sciences, Center for Integrative Infectious Disease Research (CIID), University of Heidelberg, Heidelberg, Germany.ORCID 0000-0001-6227-5665
Stephen D HauschkaSenator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA, USA.
Michael RegnierSenator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, Seattle, WA, USA.
Jeffrey S ChamberlainDepartment of Neurology, University of Washington School of Medicine, Seattle, WA, USA. jsc5@uw.edu.ORCID 0000-0001-5299-0059
Funding
Vector and Transgenic Mouse CoreP30DK017047 · NIDDK · UNIVERSITY OF WASHINGTON · PI Karin E Bornfeldt · 1986 to 2026
$41.4MWellstone Muscular Dystrophy Specialized Research Center (Seattle)P50AR065139 · NIAMS · UNIVERSITY OF WASHINGTON · PI JEFFREY S CHAMBERLAIN · 2018 to 2026
$15.9MDYSTROPHIN REPLACEMENT IN MDX MICER01AR040864 · NIAMS · UNIVERSITY OF WASHINGTON · PI JEFFREY S CHAMBERLAIN · 1991 to 2026
$8.8MUW Center for Translational Muscle Research (Overall Application)P30AR074990 · NIAMS · UNIVERSITY OF WASHINGTON · PI Jennifer Michelle Davis, DANIEL RAFTERY · 2019 to 2026
$7.5MOptimizing and validation of gene therapy vectors to treat limb girdle muscular dystophyR01NS117912 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI CHAMBERLAIN, JEFFREY S, SPENCER, MELISSA JAN · 2020 to 2024
$2.6MNIAMS NIH HHS P30 AR074990NIAMS NIH HHS P50 AR065139NIAMS NIH HHS R01 AR040864NIDDK NIH HHS P30 DK017047NINDS NIH HHS R01 NS117912
Abstract
Gene replacement using adeno-associated virus (AAV) vectors is a promising therapeutic approach for many diseases
Indexed as
DystrophinGenetic TherapyInteinsMuscular Dystrophy, DuchenneProtein SplicingAnimalsDependovirusDisease Models, AnimalGenetic VectorsHumansMaleMiceMice, Inbred mdxMuscle, SkeletalDMD protein, humanDmd protein, mouseDystrophin
Identifiers
What OpenQuestion holds
Textmetadata
LicenceTDM
Read underepoch 390
Registered trials
None linked
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.