ArticleJournal of bioanalysis & biomedicine2012
Accurate Quantitation of Dystrophin Protein in Human Skeletal Muscle Using Mass Spectrometry.
Article in Journal of bioanalysis & biomedicine, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.
What it found
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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.
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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.
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Who cites it
28 citing papers in PubMed, 49 citations in OpenAlex.
- Ataluren treatment of patients with nonsense mutation dystrophinopathy.Muscle & nerve · 2014Trial
- Mass spectrometry-based methods for biofluid biomarkers for progressive diseases: amyloid peptides and dystrophins.Bioanalysis · 2025Review
- N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy.Skeletal muscle · 2024Article
- Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.Journal of neuromuscular diseases · 2024Article
- Establishment of a Triple Quadrupole HPLC-MS Quantitation Method for Dystrophin Protein in Mouse and Human Skeletal Muscle.International journal of molecular sciences · 2023Article
- Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.Science translational medicine · 2023Article
- Dystrophin Protein Quantification as a Duchenne Muscular Dystrophy Diagnostic Biomarker in Dried Blood Spots Using Multiple Reaction Monitoring Tandem Mass Spectrometry: A Preliminary Study.Molecules (Basel, Switzerland) · 2022Article
- Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy.Journal of neuromuscular diseases · 2021Article
- Latent process model of the 6-minute walk test in Duchenne muscular dystrophy : A Bayesian approach to quantifying rare disease progression.Journal of pharmacokinetics and pharmacodynamics · 2020Article
- What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?Journal of muscle research and cell motility · 2019Review
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- Duchenne and Becker Muscular Dystrophies: A Review of Animal Models, Clinical End Points, and Biomarker Quantification.Toxicologic pathology · 2017Review
- Proceedings of the 2017 National Toxicology Program Satellite Symposium.Toxicologic pathology · 2017Article
- Somatic mosaicism due to a reversion variant causing hemi-atrophy: a novel variant of dystrophinopathy.European journal of human genetics : EJHG · 2016Article
- Neo-epitope Peptides as Biomarkers of Disease Progression for Muscular Dystrophies and Other Myopathies.Journal of neuromuscular diseases · 2016Review
- The biochemical and mass spectrometric profiling of the dystrophin complexome from skeletal muscle.Computational and structural biotechnology journal · 2016Review
- Clinical utility of serum biomarkers in Duchenne muscular dystrophy.Clinical proteomics · 2016Review
- Concurrent Label-Free Mass Spectrometric Analysis of Dystrophin Isoform Dp427 and the Myofibrosis Marker Collagen in Crude Extracts fromProteomes · 2015Article
Corrections and comments
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Authors and funding
10 authors at 1 institution in 1 country.
Funding
Abstract
Quantitation of human dystrophin protein in muscle biopsies is a clinically relevant endpoint for both diagnosis and response to dystrophin-replacement therapies for dystrophinopathies. A robust and accurate assay would enable the use of dystrophin as a surrogate biomarker, particularly in exploratory Phase 2 trials. Currently available methods to quantitate dystrophin rely on immunoblot or immunohistochemistry methods that are not considered robust. Here we present a mass spectrometry based approach to accurately quantitate dystrophin protein in a total protein extract from human muscle biopsies. Our approach uses a combination of stable isotope labeled dystrophin as a spike-in standard, gel electrophoresis and high precision mass spectrometry to detect and quantitate multiple peptides of dystrophin within a complex protein mixture. The method was found highly reproducible and linear over a wide dynamic range, detecting as low as 5% of dystrophin relative to the normal amount in healthy individuals.
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Registered trials
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.