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Article
Truncated RUNX1 protein generated by a novel t(1;21)(p32;q22) chromosomal translocation impairs the proliferation and differentiation of human hematopoietic progenitors
We have identified a new t(1;21)(p32;q22) chromosomal translocation in a MDS/AML patient that results in expression of an aberrant C-terminally truncated RUNX1 protein lacking several regulatory domains. As si...
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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...
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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...