ArticleHuman genetics1991
Human dystrophin gene transfer: production and expression of a functional recombinant DNA-based gene.
Article in Human genetics, 1991. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
What it found
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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.
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Who cites it
9 citing papers in PubMed, 43 citations in OpenAlex.
- Second-generation compound for the modulation of utrophin in the therapy of DMD.Human molecular genetics · 2015Article
- Duchenne muscular dystrophy gene therapy: Lost in translation?Research and reports in biology · 2011Article
- Immune rejection of human dystrophin following intramuscular injections of naked DNA in mdx mice.Gene therapy · 2000Article
- Enhanced expression of recombinant dystrophin following intramuscular injection of Epstein-Barr virus (EBV)-based mini-chromosome vectors in mdx mice.Gene therapy · 1999Article
- Gene therapy in surgery: Part II: Application to septic shock and to organ transplantation.Acta chirurgica Austriaca · 1997Review
- Prospects for gene therapy in sports medicine.Knee surgery, sports traumatology, arthroscopy : official journal of the ESSKA · 1996Review
- Integrated study of 100 patients with Xp21 linked muscular dystrophy using clinical, genetic, immunochemical, and histopathological data. Part 2. Correlations within individual patients.Journal of medical genetics · 1993Article
- Integrated study of 100 patients with Xp21 linked muscular dystrophy using clinical, genetic, immunochemical, and histopathological data. Part 1. Trends across the clinical groups.Journal of medical genetics · 1993Article
- Point mutations in the dystrophin gene.Proceedings of the National Academy of Sciences of the United States of America · 1992Article
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 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The identification and cloning of the gene responsible for Duchenne muscular dystrophy (DMD) and characterization of the protein product of the gene, dystrophin, has led to major advances in diagnostic and genetic counselling procedures for this inherited disorder. Due to its high mutation rate, however, individuals affected by DMD will continue to arise in large proportion by de novo mutations, and the search for direct therapies remains a high priority. In this respect direct genetic correction of dystrophin deficiency via grafting of healthy myoblast stem cells or direct introduction of functional DNA into diseased muscle tissue have both been proposed as potential therapeutic approaches. We describe here, the first example of the engineering and cloning of a synthetic gene encoding recombinant human dystrophin and its stable transfer to and expression in mammalian cells. This DMD gene construction represents a primary step towards evaluating direct DNA-mediated gene transfer as a potential treatment for this debilitating disorder.
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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.