ArticleMolecular therapy. Nucleic acids2013
Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation.
Article in Molecular therapy. Nucleic acids, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 23 citations in OpenAlex.
- Peptide-Functionalized Dendrimer Nanocarriers for Targeted Microdystrophin Gene Delivery.Pharmaceutics · 2021Article
- Genome engineering: a new approach to gene therapy for neuromuscular disorders.Nature reviews. Neurology · 2017Review
- Therapeutic gene editing: delivery and regulatory perspectives.Acta pharmacologica Sinica · 2017Review
- CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells.The Korean journal of internal medicine · 2017Review
- Engineered Viruses as Genome Editing Devices.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Review
- Genome-editing Technologies for Gene and Cell Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Review
- Neuromuscular electrical stimulation promotes development in mice of mature human muscle from immortalized human myoblasts.Skeletal muscle · 2016Article
- Advances in gene therapy for muscular dystrophies.F1000Research · 2016Review
- Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.Nature communications · 2015Article
- Adenoviral vector DNA for accurate genome editing with engineered nucleases.Nature methods · 2014Article
- Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells.Frontiers in physiology · 2014Review
Corrections and comments
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Authors and funding
8 authors at 5 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Zinc finger nucleases (ZFN) can facilitate targeted gene addition to the genome while minimizing the risks of insertional mutagenesis. Here, we used a previously characterized ZFN pair targeting the chemokine (C-C motif) receptor 5 (CCR5) locus to introduce, as a proof of concept, the enhanced green fluorescent protein (eGFP) or the microdystrophin genes into human myoblasts. Using integrase-defective lentiviral vectors (IDLVs) and chimeric adenoviral vectors to transiently deliver template DNA and ZFN respectively, we achieved up to 40% targeted gene addition in human myoblasts. When the O(6)-methylguanine-DNA methyltransferase(P140K) gene was co-introduced with eGFP, the frequency of cells with targeted integration could be increased to over 90% after drug selection. Importantly, gene-targeted myoblasts retained their mitogenic activity and potential to form myotubes both in vitro and in vivo when injected into the tibialis anterior of immune-deficient mice. Altogether, our results could lead to the development of improved cell therapy transplantation protocols for muscular diseases.Molecular Therapy - Nucleic Acids (2013) 2, e68; doi:10.1038/mtna.2012.55; published online 29 January 2013.
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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.