ReviewCurrent opinion in neurology2009
Emerging genetic therapies to treat Duchenne muscular dystrophy.
Review in Current opinion in neurology, 2009. 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
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
28 citing papers in PubMed, 72 citations in OpenAlex.
- Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.PloS one · 2013Trial
- Antisense oligonucleotide: A promising therapeutic option to beat COVID-19.Wiley interdisciplinary reviews. RNA · 2022Review
- Plant Antimicrobial Peptides as Potential Tool for Topic Treatment of Hidradenitis Suppurativa.Frontiers in microbiology · 2021Review
- The position of nonsense mutations can predict the phenotype severity: A survey on the DMD gene.PloS one · 2020Article
- Neurons under genetic control: What are the next steps towards the treatment of movement disorders?Computational and structural biotechnology journal · 2020Review
- Functional Mixed-Effects Modeling of Longitudinal Duchenne Muscular Dystrophy Electrical Impedance Myography Data Using State-Space Approach.IEEE transactions on bio-medical engineering · 2019Article
- Antisense oligonucleotides: A primer.Neurology. Genetics · 2019Review
- Oligonucleotide therapeutics in neurodegenerative diseases.RNA biology · 2018Review
- Nutraceuticals and Their Potential to Treat Duchenne Muscular Dystrophy: Separating the Credible from the Conjecture.Nutrients · 2016Review
- Composite biomarkers for assessing Duchenne muscular dystrophy: an initial assessment.Pediatric neurology · 2015Article
- Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients.Human molecular genetics · 2014Article
- Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.American journal of physical medicine & rehabilitation · 2014Review
- Parathyroid hormone and parathyroid hormone type-1 receptor accelerate myocyte differentiation.Scientific reports · 2014Article
- Identification and characterization of small molecules that inhibit nonsense-mediated RNA decay and suppress nonsense p53 mutations.Cancer research · 2014Article
- Electrical impedance myography for the assessment of children with muscular dystrophy: a preliminary study.Journal of physics. Conference series · 2013Article
- Accurate Quantitation of Dystrophin Protein in Human Skeletal Muscle Using Mass Spectrometry.Journal of bioanalysis & biomedicine · 2012Article
- Sarcopenia: pharmacology of today and tomorrow.The Journal of pharmacology and experimental therapeutics · 2012Review
- Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2012Review
- The correlation analysis of functional factors and age with duchenne muscular dystrophy.Annals of rehabilitation medicine · 2012Article
- Control of gene expression by translational recoding.Advances in protein chemistry and structural biology · 2012Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 3 institutions in 1 country.
Funding
Abstract
purpose of reviewDuchenne muscular dystrophy is a progressive muscle degenerative disease caused by dystrophin mutations. The purpose of this review is to highlight two emerging therapies designed to repair the primary genetic defect, called 'exon skipping' and 'nonsense codon suppression'. RECENT
findingsA drug, PTC124, was identified that suppresses nonsense codon translation termination. PTC124 can lead to restoration of some dystrophin expression in human Duchenne muscular dystrophy muscles with mutations resulting in premature stops. Two drugs developed for exon skipping, PRO051 and AVI-4658, result in the exclusion of exon 51 from mature mRNA. They can restore the translational reading frame to dystrophin transcripts from patients with a particular subset of dystrophin gene deletions and lead to some restoration of dystrophin expression in affected boys' muscle in vivo. Both approaches have concluded phase I trials with no serious adverse events. SUMMARY: These novel therapies that act to correct the primary genetic defect of dystrophin deficiency are among the first generation of therapies tailored to correct specific mutations in humans. Thus, they represent paradigm forming approaches to personalized medicine with the potential to lead to life changing treatment for those affected by Duchenne muscular dystrophy.
Indexed as
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What OpenQuestion holds
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.