Successful low-risk hematopoietic cell therapy in a mouse model of type 1 Gaucher disease.

  title={Successful low-risk hematopoietic cell therapy in a mouse model of type 1 Gaucher disease.},
  author={Ida Berglin Enquist and Eva Falk Nilsson and J -E Mansson and Mats Ehinger and Johan C O Richter and Stefan Karlsson},
  journal={Stem cells},
  volume={27 3},
Hematopoietic stem cell-based gene therapy offers the possibility of permanent correction for genetic disorders of the hematopoietic system. However, optimization of present protocols is required before gene therapy can be safely applied as general treatment of genetic diseases. In this study we have used a mouse model of type 1 Gaucher disease (GD) to demonstrate the feasibility of a low-risk conditioning regimen instead of standard radiation, which is associated with severe adverse effects… CONTINUE READING


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