ReviewGene therapy2006
Gene therapy progress and prospects: Duchenne muscular dystrophy.
Review in Gene therapy, 2006. 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, 74 citations in OpenAlex.
- Episomal minicircles persist in periods of transcriptional inactivity and can be transmitted through somatic cell nuclear transfer into bovine embryos.Molecular biology reports · 2019Article
- Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice.Molecular therapy. Nucleic acids · 2017Article
- Therapeutic effects of long-circulating miR-135a-containing cationic immunoliposomes against gallbladder carcinoma.Scientific reports · 2017Article
- Evaluation of Amphiphilic Peptide Modified Antisense Morpholino Oligonucleotides In Vitro and in Dystrophic mdx Mice.Polymers · 2017Article
- Poly(ester amine) Composed of Polyethylenimine and Pluronic Enhance Delivery of Antisense Oligonucleotides In Vitro and in Dystrophic mdx Mice.Molecular therapy. Nucleic acids · 2016Article
- The pharmacology of regenerative medicine.Pharmacological reviews · 2013Review
- Polyethylenimine-modified pluronics (PCMs) improve morpholino oligomer delivery in cell culture and dystrophic mdx mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
- Capsid-modified adenoviral vectors for improved muscle-directed gene therapy.Human gene therapy · 2012Article
- Thermodynamic stability, unfolding kinetics, and aggregation of the N-terminal actin-binding domains of utrophin and dystrophin.Proteins · 2012Article
- Engraftment of ES-Derived Myogenic Progenitors in a Severe Mouse Model of Muscular Dystrophy.Journal of stem cell research & therapy · 2012Article
- Rescue of dystrophic skeletal muscle by PGC-1α involves a fast to slow fiber type shift in the mdx mouse.PloS one · 2012Article
- One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Article
- Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.Cardiovascular research · 2010Article
- Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino.Gene therapy · 2010Article
- Femtosecond laser treatment enhances DNA transfection efficiency in vivo.Journal of biomedical science · 2009Article
- A highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin gene.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Cardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Gene diagnosis for nine Chinese patients with DMD/BMD by multiplex ligation-dependent probe amplification and prenatal diagnosis for one of them.Journal of clinical laboratory analysis · 2009Article
- Gene therapy in large animal models of muscular dystrophy.ILAR journal · 2009Review
- Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer.Proceedings of the National Academy of Sciences of the United States of America · 2008Article
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
No grant is acknowledged in the PubMed record.
Abstract
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder affecting 1/3500 male births. There is currently no effective treatment, but gene therapy approaches are offering viable avenues for treatment development. The last 10 years have seen the development of a number of strategies and tools for muscle gene therapy. However, the major hurdle has been the inability to deliver vectors at high enough efficiency via a systemic route. The last 2-3 years (reviewed here) have seen unrivalled progress in efficient systemic delivery of viral and non-viral gene transfer agents and antisense oligonucleotides. This progress, coupled with the successful completion of the first gene therapy clinical trial for DMD, has led to three more clinical trials planned for the immediate future.
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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.