ArticleGene therapy2003
Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer.
Article in Gene therapy, 2003. 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, 117 citations in OpenAlex.
- Leading edge: emerging drug, cell, and gene therapies for junctional epidermolysis bullosa.Expert opinion on biological therapy · 2020Review
- Preclinical and clinical advances in transposon-based gene therapy.Bioscience reports · 2017Review
- Next generation human skin constructs as advanced tools for drug development.Experimental biology and medicine (Maywood, N.J.) · 2017Review
- From marrow to matrix: novel gene and cell therapies for epidermolysis bullosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Review
- Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Advances in understanding and treating dystrophic epidermolysis bullosa.F1000prime reports · 2014Review
- Novel therapeutic approaches for various cancer types using a modified sleeping beauty-based gene delivery system.PloS one · 2014Article
- DNA transposon-based gene vehicles - scenes from an evolutionary drive.Journal of biomedical science · 2013Review
- AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo.Human gene therapy · 2012Article
- Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies.Current gene therapy · 2011Review
- Keratin gene mutations in disorders of human skin and its appendages.Archives of biochemistry and biophysics · 2011Review
- The Sleeping Beauty transposon system: a non-viral vector for gene therapy.Human molecular genetics · 2011Review
- Efficient non-viral integration and stable gene expression in multipotent adult progenitor cells.Stem cells international · 2011Article
- Increased interstitial pressure improves nucleic acid delivery to skin enabling a comparative analysis of constitutive promoters.Gene therapy · 2010Article
- Translating Sleeping Beauty transposition into cellular therapies: victories and challenges.BioEssays : news and reviews in molecular, cellular and developmental biology · 2010Review
- Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.Hepatology (Baltimore, Md.) · 2010Article
- A transposon and transposase system for human application.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Review
- Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons.Human gene therapy · 2010Article
- Efficient nonviral Sleeping Beauty transposon-based TCR gene transfer to peripheral blood lymphocytes confers antigen-specific antitumor reactivity.Gene therapy · 2009Article
- Hybrid lentivirus-transposon vectors with a random integration profile in human cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sustainable correction of severe human genetic disorders of self-renewing tissues, such as the blistering skin disease junctional epidermolysis bullosa (JEB), is facilitated by stable genomic integration of therapeutic genes into somatic tissue stem cells. While integrating viral vectors can achieve this, they suffer from logistical and biosafety concerns. To circumvent these limitations, we used the Sleeping Beauty transposable element to integrate the LAMB3 cDNA into genomes of epidermal holoclones from six unrelated JEB patients. These cells regenerate human JEB skin that is normalized at the level of laminin 5 protein expression, hemidesmosome formation and blistering. Transposon-mediated gene delivery therefore affords an opportunity for stable gene delivery in JEB and other human diseases.
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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.