ArticleGene therapy2006
Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies.
Article in Gene therapy, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed, 33 citations in OpenAlex.
- Impact of downstream purification process and AAV serotype on protein-impurity clearance.Molecular therapy. Advances · 2026Article
- Article
- AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression.Human molecular genetics · 2017Article
- Cryosectioning of Contiguous Regions of a Single Mouse Skeletal Muscle for Gene Expression and Histological Analyses.Journal of visualized experiments : JoVE · 2016Article
- Rapid, scalable, and low-cost purification of recombinant adeno-associated virus produced by baculovirus expression vector system.Molecular therapy. Methods & clinical development · 2016Article
- Simple downstream process based on detergent treatment improves yield and in vivo transduction efficacy of adeno-associated virus vectors.Molecular therapy. Methods & clinical development · 2015Article
- A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence.Molecular therapy. Methods & clinical development · 2015Article
- Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Pancreatic transduction by helper-dependent adenoviral vectors via intraductal delivery.Human gene therapy · 2014Article
- Full-length dystrophin reconstitution with adeno-associated viral vectors.Human gene therapy · 2014Article
- Limb-girdle muscular dystrophies: where next after six decades from the first proposal (Review).Molecular medicine reports · 2014Review
- Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.Nucleic acids research · 2013Article
- AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
- Transduction of skeletal muscles with common reporter genes can promote muscle fiber degeneration and inflammation.PloS one · 2012Article
- The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
- Real-time monitoring of cell transplantation in mouse dystrophic muscles by a secreted alkaline phosphatase reporter gene.Gene therapy · 2009Article
- Concordant activity of transgene expression cassettes inserted into E1, E3 and E4 cloning sites in the adenovirus genome.The journal of gene medicine · 2009Article
- AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not alpha-sarcoglycan deficiency.Gene therapy · 2007Article
- Splicing analysis disclosed a determinant single nucleotide for exon skipping caused by a novel intraexonic four-nucleotide deletion in the dystrophin gene.Journal of medical genetics · 2006Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Muscular dystrophies are a genetically and phenotypically heterogeneous group of degenerative muscle diseases. A subset of them are due to genetic deficiencies in proteins which form the dystrophin-associated complex at the membrane of the myofibers. In this report, we utilized recombinant adeno-associated virus containing a U7 cassette carrying an antisense sequence aimed at inducing exon skipping of the dystrophin gene or containing the alpha-sarcoglycan gene to alleviate the dystrophic phenotype of the mdx and Sgca-null mice, respectively. As these diseases are characterized by cycle of degeneration/regeneration, we postulated that a reporter gene coadministered at the time of the treatment would make it possible to follow the extent of muscle repair. We observed that the murine secreted alkaline phosphatase (muSeAP) level was very much lower in these animal models than in normal mice. Upon treatment of the dystrophic muscle by gene transfer, the level of muSeAP was restored and correlated with the expression of the therapeutic transgene and with the level of muscle improvement. The system described here provides a simple and noninvasive procedure for monitoring the outcome of a therapeutic strategy involving cell survival.
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.