TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells.

@article{Dreyer2015TALENmediatedFC,
  title={TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells.},
  author={Anne-Kathrin Dreyer and Dirk Hoffmann and Nico Lachmann and Mathias Ackermann and Doris Steinemann and Barbara K Timm and Ulrich Siler and Janine Reichenbach and Manuel Grez and Thomas Moritz and Michellle Cross and Toni Cathomen},
  journal={Biomaterials},
  year={2015},
  volume={69},
  pages={
          191-200
        }
}
X-linked chronic granulomatous disease (X-CGD) is an inherited disorder of the immune system. It is characterized by a defect in the production of reactive oxygen species (ROS) in phagocytic cells due to mutations in the NOX2 locus, which encodes gp91phox. Because the success of retroviral gene therapy for X-CGD has been hampered by insertional activation of proto-oncogenes, targeting the insertion of a gp91phox transgene into potential safe harbor sites, such as AAVS1, may represent a valid… CONTINUE READING
BETA

Similar Papers

Citations

Publications citing this paper.
SHOWING 1-10 OF 17 CITATIONS

References

Publications referenced by this paper.
SHOWING 1-8 OF 8 REFERENCES

Lentiviral vector design and imaging approaches to visualize the early stages of cellular reprogramming.

  • Molecular therapy : the journal of the American Society of Gene Therapy
  • 2011
VIEW 1 EXCERPT