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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
Site-specific gene addition can allow stable transgene expression for gene therapy. When possible, this is preferred over the use of promiscuously integrating vectors, which are sometimes associated with clonal expansion(1) and oncogenesis(2). Site-specific endonucleases that can induce high rates o...
| Autores principales: | , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
2014
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4297598/ https://www.ncbi.nlm.nih.gov/pubmed/25363772 http://dx.doi.org/10.1038/nature13864 |