ReviewAmerican journal of physical medicine & rehabilitation2014
Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.
Review in American journal of physical medicine & rehabilitation, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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
14 citing papers in PubMed, 26 citations in OpenAlex.
- Biochemical Changes in Blood of Patients with Duchenne Muscular Dystrophy Treated with Granulocyte-Colony Stimulating Factor.BioMed research international · 2019Trial
- Regenerative Medicine: Advanced Therapy for Muscle Tissue Restoration.International journal of molecular sciences · 2026Review
- Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders.Neurological research and practice · 2022Review
- Review
- Duchenne's Muscular Dystrophy: The Role of Induced Pluripotent Stem Cells and Genomic Editing on Muscle Regeneration.Cureus · 2020Review
- Current understanding of and emerging treatment options for spinal muscular atrophy with respiratory distress type 1 (SMARD1).Cellular and molecular life sciences : CMLS · 2020Review
- Photobiomodulation promotes adenoviral gene transduction in auditory cells.Lasers in medical science · 2019Article
- Antisense oligonucleotides in neurological disorders.Therapeutic advances in neurological disorders · 2018Review
- Pharmacological inhibition of REV-ERB stimulates differentiation, inhibits turnover and reduces fibrosis in dystrophic muscle.Scientific reports · 2017Article
- Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies.Scientific reports · 2016Article
- Human induced pluripotent stem cells for monogenic disease modelling and therapy.World journal of stem cells · 2016Review
- Stem Cell Differentiation Toward the Myogenic Lineage for Muscle Tissue Regeneration: A Focus on Muscular Dystrophy.Stem cell reviews and reports · 2015Review
- Clinical and molecular features and therapeutic perspectives of spinal muscular atrophy with respiratory distress type 1.Journal of cellular and molecular medicine · 2015Review
- Duchenne muscular dystrophy: current cell therapies.Therapeutic advances in neurological disorders · 2015Review
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 2 institutions in 2 countries.
Funding
Abstract
The development of clinical vectors to correct genetic mutations that cause inherited myopathies and related disorders of skeletal muscle is advancing at an impressive rate. Adeno-associated virus vectors are attractive for clinical use because (1) adeno-associated viruses do not cause human disease and (2) these vectors are able to persist for years. New vectors are now becoming available as gene therapy delivery tools, and recent preclinical experiments have demonstrated the feasibility, safety, and efficacy of gene therapy with adeno-associated virus for long-term correction of muscle pathology and weakness in myotubularin-deficient canine and murine disease models. In this review, recent advances in the application of gene therapies to treat inherited muscle disorders are presented, including Duchenne muscular dystrophy and x-linked myotubular myopathy. Potential areas for therapeutic synergies between rehabilitation medicine and genetics are also discussed.
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Identifiers
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.