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  1. No Access

    Article

    An integration-defective lentivirus-based resource for site-specific targeting of an edited safe-harbour locus in the human genome

    Optimized gene transfer into human cells are still challenging the promise of human stem and induced pluripotent stem cells as resources for disease models, diagnostic screens and personalized cell therapy. Th...

    R Torres, A Garcia, M Jimenez, S Rodriguez, J C Ramirez in Gene Therapy (2014)

  2. No Access

    Article

    Immunoresponse against the transgene limits hematopoietic engraftment of mice transplanted in utero with virally transduced fetal liver

    In utero cell and gene therapies constitute alternative strategies to the postnatal treatment of inherited diseases. Fetal hematopoietic progenitors could be a potential source of donor cells for these strategies...

    M E Alonso-Ferrero, A Valeri, R Yañez, S Navarro, M I Garin, J C Ramirez… in Gene Therapy (2011)