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...

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Detalles Bibliográficos
Autores principales: Barzel, A., Paulk, N.K., Shi, Y., Huang, Y., Chu, K., Zhang, F., Valdmanis, P.N., Spector, L.P., Porteus, M.H., Gaensler, K.M., Kay, M.A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2014
Materias:
Article
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

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4297598/
https://www.ncbi.nlm.nih.gov/pubmed/25363772
http://dx.doi.org/10.1038/nature13864

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