ReviewBiochimica et biophysica acta2007
Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances.
Review in Biochimica et biophysica acta, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 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
37 citing papers in PubMed, 96 citations in OpenAlex.
- Putting the "mi" in omics: discovering miRNA biomarkers for pediatric precision care.Pediatric research · 2023Review
- Duchenne muscular dystrophy: disease mechanism and therapeutic strategies.Frontiers in physiology · 2023Review
- Effects of low-intensity training on the brain and muscle in the congenital muscular dystrophy 1D model.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2022Article
- Highly Efficient Microcell-Mediated Transfer of HACs Containing a Genomic Region of Interest into Mammalian Cells.Current protocols · 2021Article
- Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy.Proceedings of the National Academy of Sciences of the United States of America · 2018Article
- Molecular Therapies for Muscular Dystrophies.Current treatment options in neurology · 2018Review
- Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy.Expert opinion on orphan drugs · 2016Article
- Reduction in mdx mouse muscle degeneration by low-intensity endurance exercise: a proteomic analysis in quadriceps muscle of exercised compared with sedentary mdx mice.Bioscience reports · 2015Article
- A pathway from chromosome transfer to engineering resulting in human and mouse artificial chromosomes for a variety of applications to bio-medical challenges.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2015Review
- Para amino benzoic acid-derived self-assembled biocompatible nanoparticles for efficient delivery of siRNA.International journal of nanomedicine · 2015Article
- Efficient in vitro gene delivery by hybrid biopolymer/virus nanobiovectors.Journal of controlled release : official journal of the Controlled Release Society · 2014Article
- Article
- Design of hybrid lipid/retroviral-like particle gene delivery vectors.Molecular pharmaceutics · 2013Article
- Re-engineering an alphoid(tetO)-HAC-based vector to enable high-throughput analyses of gene function.Nucleic acids research · 2013Article
- A new generation of human artificial chromosomes for functional genomics and gene therapy.Cellular and molecular life sciences : CMLS · 2013Review
- Cellular Transplantation Alters the Disease Progression in Becker's Muscular Dystrophy.Case reports in transplantation · 2013Article
- Thermodynamic stability, unfolding kinetics, and aggregation of the N-terminal actin-binding domains of utrophin and dystrophin.Proteins · 2012Article
- β1D chain increases α7β1 integrin and laminin and protects against sarcolemmal damage in mdx mice.Human molecular genetics · 2012Article
- AAV-microdystrophin therapy improves cardiac performance in aged female mdx mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Human artificial chromosomes for gene delivery and the development of animal models.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
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
Much progress has been made over the past decade elucidating the molecular basis for a variety of muscular dystrophies (MDs). Accordingly, there are examples of mouse models of MD whose disease progression has been halted in large part with the use of viral vector technology. Even so, we must acknowledge significant limitations of present vector systems that must be overcome prior to successful treatment of humans with such approaches. This review will present a variety of viral-mediated therapeutic strategies aimed at counteracting the muscle-wasting symptoms associated with muscular dystrophy. We include viral vector systems used for muscle gene transfer, with a particular emphasis on adeno-associated virus. Findings of several encouraging studies focusing on repair of the mutant dystrophin gene are also included. Lastly, we present a discussion of muscle compensatory therapeutics being considered that include pathways involved in the up-regulation of utrophin, promotion of cellular adhesion, enhancement of muscle mass, and antagonism of the inflammatory response. Considering the complexity of the muscular dystrophies, it appears likely that a multilayered approach tailored to a patient sub-group may be warranted in order to effectively contest the progression of this devastating disease.
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Identifiers
What Socratic 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 Socratic graph.