ArticleNucleic acids research2011
Native homing endonucleases can target conserved genes in humans and in animal models.
Article in Nucleic acids research, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed, 27 citations in OpenAlex.
- Group I introns: Structure, splicing and their applications in medical mycology.Genetics and molecular biology · 2024Article
- Neighboring inteins interfere with one another's homing capacity.PNAS nexus · 2023Article
- Genome editing and the next generation of antiviral therapy.Human genetics · 2016Review
- Analysis of the Mitochondrial Genome in Hypomyces aurantius Reveals a Novel Twintron Complex in Fungi.International journal of molecular sciences · 2016Article
- Massively parallel determination and modeling of endonuclease substrate specificity.Nucleic acids research · 2014Article
- Article
- The design and in vivo evaluation of engineered I-OnuI-based enzymes for HEG gene drive.PloS one · 2013Article
- The brown algae Pl.LSU/2 group II intron-encoded protein has functional reverse transcriptase and maturase activities.PloS one · 2013Article
- LAHEDES: the LAGLIDADG homing endonuclease database and engineering server.Nucleic acids research · 2012Article
- Home and away- the evolutionary dynamics of homing endonucleases.BMC evolutionary biology · 2011Article
- Mining endonuclease cleavage determinants in genomic sequence data.The Journal of biological chemistry · 2011Article
- Tapping natural reservoirs of homing endonucleases for targeted gene modification.Proceedings of the National Academy of Sciences of the United States of America · 2011Article
- Targeted gene therapies: tools, applications, optimization.Critical reviews in biochemistry and molecular biologyReview
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Authors and funding
10 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In recent years, both homing endonucleases (HEases) and zinc-finger nucleases (ZFNs) have been engineered and selected for the targeting of desired human loci for gene therapy. However, enzyme engineering is lengthy and expensive and the off-target effect of the manufactured endonucleases is difficult to predict. Moreover, enzymes selected to cleave a human DNA locus may not cleave the homologous locus in the genome of animal models because of sequence divergence, thus hampering attempts to assess the in vivo efficacy and safety of any engineered enzyme prior to its application in human trials. Here, we show that naturally occurring HEases can be found, that cleave desirable human targets. Some of these enzymes are also shown to cleave the homologous sequence in the genome of animal models. In addition, the distribution of off-target effects may be more predictable for native HEases. Based on our experimental observations, we present the HomeBase algorithm, database and web server that allow a high-throughput computational search and assignment of HEases for the targeting of specific loci in the human and other genomes. We validate experimentally the predicted target specificity of candidate fungal, bacterial and archaeal HEases using cell free, yeast and archaeal assays.
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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.