ReviewPediatric neurology2014
Gene therapy for muscular dystrophy: moving the field forward.
Review in Pediatric neurology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.
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
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 44 citations in OpenAlex.
- Genomic regions associated with muscularity in beef cattle differ in five contrasting cattle breeds.Genetics, selection, evolution : GSE · 2020Pooled it
- In Vivo DYSF Gene Viral Delivery Provides a Histoprotective Effect in Skeletal Muscle Tissue in Dysferlin-Deficient Mice.Bulletin of experimental biology and medicine · 2023Article
- Current Clinical Applications of In Vivo Gene Therapy with AAVs.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- A well-tolerated core needle muscle biopsy process suitable for children and adults.Muscle & nerve · 2020Article
- Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine.Expert opinion on drug discovery · 2020Review
- Population-Wide Duchenne Muscular Dystrophy Carrier Detection by CK and Molecular Testing.BioMed research international · 2020Article
- Effect of AAV9-hIGF-1 on inflammatory reaction in mdx mice and its mechanism.American journal of translational research · 2020Article
- Regulation of fibrosis in muscular dystrophy.Matrix biology : journal of the International Society for Matrix Biology · 2018Review
- Current and Emerging Therapies for Duchenne Muscular Dystrophy.Current treatment options in neurology · 2018Review
- Genome engineering: a new approach to gene therapy for neuromuscular disorders.Nature reviews. Neurology · 2017Review
- A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy.Molecular therapy. Methods & clinical development · 2017Article
- Homozygous nonsense mutation in SGCA is a common cause of limb-girdle muscular dystrophy in Assiut, Egypt.Muscle & nerve · 2016Article
- Genomic removal of a therapeutic mini-dystrophin gene from adult mice elicits a Duchenne muscular dystrophy-like phenotype.Human molecular genetics · 2016Article
- [Research progress on disease models and gene therapy of Duchenne muscular dystrophy].Zhejiang da xue xue bao. Yi xue ban = Journal of Zhejiang University. Medical sciences · 2016Review
- Health-related quality of life and functional changes in DMD: A 12-month longitudinal cohort study.Neuromuscular disorders : NMD · 2016Article
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.Science (New York, N.Y.) · 2016Article
- Current and emerging treatment strategies for Duchenne muscular dystrophy.Neuropsychiatric disease and treatment · 2016Review
- Stem Cell Differentiation Toward the Myogenic Lineage for Muscle Tissue Regeneration: A Focus on Muscular Dystrophy.Stem cell reviews and reports · 2015Review
- Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model.Science advances · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Gene therapy for the muscular dystrophies has evolved as a promising treatment for this progressive group of disorders. Although corticosteroids and/or supportive treatments remain the standard of care for Duchenne muscular dystrophy, loss of ambulation, respiratory failure, and compromised cardiac function is the inevitable outcome. Recent developments in genetically mediated therapies have allowed for personalized treatments that strategically target individual muscular dystrophy subtypes based on disease pathomechanism and phenotype. In this review, we highlight the therapeutic progress with emphasis on evolving preclinical data and our own experience in completed clinical trials and others currently underway. We also discuss the lessons we have learned along the way and the strategies developed to overcome limitations and obstacles in this field.
Indexed as
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.