ArticleGene therapy2002
Simultaneous targeted alteration of the tyrosinase and c-kit genes by single-stranded oligonucleotides.
Article in Gene therapy, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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Who cites it
18 citing papers in PubMed, 45 citations in OpenAlex.
- Recent advances in Duchenne muscular dystrophy.Degenerative neurological and neuromuscular disease · 2012Review
- An update on targeted gene repair in mammalian cells: methods and mechanisms.Journal of biomedical science · 2011Review
- A comparison of synthetic oligodeoxynucleotides, DNA fragments and AAV-1 for targeted episomal and chromosomal gene repair.BMC biotechnology · 2009Article
- Site-specific gene modification by oligodeoxynucleotides in mouse bone marrow-derived mesenchymal stem cells.Gene therapy · 2008Article
- Oligonucleotide-mediated gene targeting in human hepatocytes: implications of mismatch repair.Oligonucleotides · 2008Article
- Single-stranded oligonucleotide-mediated in vivo gene repair in the rd1 retina.Molecular vision · 2007Article
- Oligonucleotide-directed gene repair in wheat using a transient plasmid gene repair assay system.Plant cell reports · 2006Article
- Site-specific base changes in the coding or promoter region of the human beta- and gamma-globin genes by single-stranded oligonucleotides.The Biochemical journal · 2005Article
- Gene therapy progress and prospects: targeted gene repair.Gene therapy · 2005Review
- Implications of cell cycle progression on functional sequence correction by short single-stranded DNA oligonucleotides.Gene therapy · 2005Article
- Increased efficiency of oligonucleotide-mediated gene repair through slowing replication fork progression.Proceedings of the National Academy of Sciences of the United States of America · 2005Article
- Stable transmission of targeted gene modification using single-stranded oligonucleotides with flanking LNAs.Nucleic acids research · 2005Article
- Site-specific strand bias in gene correction using single-stranded oligonucleotides.Journal of molecular medicine (Berlin, Germany) · 2005Article
- Ultraviolet light selection assay to optimize oligonucleotide correction of mutations in endogenous xeroderma pigmentosum genes.Gene therapy · 2004Article
- A LacZ-based transgenic mouse for detection of somatic gene repair events in vivo.Gene therapy · 2004Article
- Transcription affects formation and processing of intermediates in oligonucleotide-mediated gene alteration.Nucleic acids research · 2003Article
- Expectations and reality in gene repair.Nature biotechnology · 2002Article
- Review
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
We have shown that various forms of oligonucleotides, chimeric RNA-DNA oligonucleotide (RDO) and single-stranded oligodeoxynucleotide (ODN), are capable of chromosomal gene alterations in mammalian cells. Using two ODNs we corrected an inactivating mutation in the tyrosinase gene and introduced an activating mutation into the c-kit gene in a single albino mouse melanocyte. Relying on a pigmentation change caused by tyrosinase gene correction, we determined the frequency of gene targeting events ranging from 2 x 10(-4) to 1 x 10(-3), which is comparable to our previously published data using RDO. However, ODN showed more reproducible gene correction than RDO and produced pigmented cells among 60% of experiments, in comparison with 10% by RDO. DNA sequence analysis of the converted cells revealed that two out of eight individual pigmented clones harbored the mutated c-kit gene. Targeted modification of both genes resulted in the ability of the tyrosinase to convert tyrosine to melanin, and in the constitutive activation of the Kit receptor kinase. Thus, for the first time, we demonstrate the feasibility of simultaneous targeting of two genes in a single cell and show that a selection strategy to identify cells that have undergone a gene modification can enrich the targeted cells with the desired gene alteration.
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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.