ArticleJournal of virology2005
Transfer of the full-length dystrophin-coding sequence into muscle cells by a dual high-capacity hybrid viral vector with site-specific integration ability.
Article in Journal of virology, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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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
16 citing papers in PubMed, 44 citations in OpenAlex.
- Review
- High-Capacity Adenoviral Vectors: Expanding the Scope of Gene Therapy.International journal of molecular sciences · 2020Review
- Adenovirus vectors in hematopoietic stem cell genome editing.FEBS letters · 2019Review
- Inhibition of antigen presentation during AAV gene therapy using virus peptides.Human molecular genetics · 2018Article
- Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.American journal of translational research · 2016Review
- Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery.Human gene therapy methods · 2014Article
- Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
- Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.Pharmaceuticals (Basel, Switzerland) · 2013Article
- Concerted nicking of donor and chromosomal acceptor DNA promotes homology-directed gene targeting in human cells.Nucleic acids research · 2012Article
- Integration preferences of wildtype AAV-2 for consensus rep-binding sites at numerous loci in the human genome.PLoS pathogens · 2010Article
- Stimulation of homology-directed gene targeting at an endogenous human locus by a nicking endonuclease.Nucleic acids research · 2009Article
- Targeted chromosomal insertion of large DNA into the human genome by a fiber-modified high-capacity adenovirus-based vector system.PloS one · 2008Article
- Adeno-associated virus integration: virus versus vector.Gene therapy · 2008Review
- Integration of active human beta-galactosidase gene (100 kb) into genome using HSV/AAV amplicon vector.Gene therapy · 2007Article
- Therapeutics in duchenne muscular dystrophy.NeuroRx : the journal of the American Society for Experimental NeuroTherapeutics · 2006Review
- Gutless adenovirus: last-generation adenovirus for gene therapy.Gene therapy · 2005Review
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, making it a potential target for gene therapy. There is, however, a scarcity of vectors that can accommodate the 14-kb DMD cDNA and permanently genetically correct muscle tissue in vivo or proliferating myogenic progenitors in vitro for use in autologous transplantation. Here, a dual high-capacity adenovirus-adeno-associated virus (hcAd/AAV) vector with two full-length human dystrophin-coding sequences flanked by AAV integration-enhancing elements is presented. These vectors are generated from input linear monomeric DNA molecules consisting of the Ad origin of replication and packaging signal followed by the recently identified AAV DNA integration efficiency element (p5IEE), the transgene(s) of interest, and the AAV inverted terminal repeat (ITR). After infection of producer cells with a helper Ad vector, the Ad DNA replication machinery, in concert with the AAV ITR-dependent dimerization, leads to the assembly of vector genomes with a tail-to-tail configuration that are efficiently amplified and packaged into Ad capsids. These dual hcAd/AAV hybrid vectors were used to express the dystrophin-coding sequence in rat cardiomyocytes in vitro and to restore dystrophin synthesis in the muscle tissues of mdx mice in vivo. Introduction into human cells of chimeric genomes, which contain a structure reminiscent of AAV proviral DNA, resulted in AAV Rep-dependent targeted DNA integration into the AAVS1 locus on chromosome 19. Dual hcAd/AAV hybrid vectors may thus be particularly useful to develop safe treatment modalities for diseases such as DMD that rely on efficient transfer and stable expression of large genes.
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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.