ArticleGene therapy2004
Long-term expression of full-length human dystrophin in transgenic mdx mice expressing internally deleted human dystrophins.
Article in Gene therapy, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 34 citations in OpenAlex.
- Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.The Lancet. Neurology · 2009Trial
- From Mice to Humans: An Overview of the Potentials and Limitations of Current Transgenic Mouse Models of Major Muscular Dystrophies and Congenital Myopathies.International journal of molecular sciences · 2020Review
- 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
- Multiplicity of experimental approaches to therapy for genetic muscle diseases and necessity for population screening.Journal of muscle research and cell motility · 2008Review
- Molecular-targeted therapy for Duchenne muscular dystrophy: progress and potential.Molecular diagnosis & therapy · 2008Review
- Gene therapy progress and prospects: Duchenne muscular dystrophy.Gene therapy · 2006Review
- Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy.Journal of muscle research and cell motility · 2006Review
- Nonviral gene transfer to skeletal, smooth, and cardiac muscle in living animals.American journal of physiology. Cell physiology · 2005Review
- DNA electrotransfer: its principles and an updated review of its therapeutic applications.Gene therapy · 2004Review
- Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD).Gene therapy · 2004Review
- Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment.EMBO reports · 2004Review
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
One of the possible therapies for Duchenne muscular dystrophy (DMD) is the introduction of a functional copy of the dystrophin gene into the patient. For this approach to be effective, therapeutic levels and long-term expression of the protein need to be achieved. However, immune responses to the newly expressed dystrophin have been predicted, particularly in DMD patients who express no dystrophin or only very truncated versions. In a previous study, we demonstrated a strong humoral and cytotoxic immune response to human dystrophin in the mdx mouse. However, the mdx mouse was tolerant to murine dystrophin, possibly due to the endogenous expression of dystrophin in revertant fibres or the other nonmuscle dystrophin isoforms. In the present study, we delivered human and murine dystrophin plasmids by electrotransfer after hyaluronidase pretreatment to increase gene transfer efficiencies. Tolerance to murine dystrophin was still seen with this improved gene delivery. Tolerance to exogenous recombinant full-length human dystrophin was seen in mdx transgenic lines expressing internally deleted versions of human dystrophin. These results suggest that the presence of revertant fibres may prevent the development of serious immune responses in patients undergoing dystrophin gene therapy.
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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.