Potential large animal models for gene therapy of human genetic diseases of immune and blood cell systems.

@article{Bauer2009PotentialLA,
  title={Potential large animal models for gene therapy of human genetic diseases of immune and blood cell systems.},
  author={Thomas R Bauer and Rima L Adler and Dennis Durand Hickstein},
  journal={ILAR journal},
  year={2009},
  volume={50 2},
  pages={168-86}
}
Genetic mutations involving the cellular components of the hematopoietic system--red blood cells, white blood cells, and platelets--manifest clinically as anemia, infection, and bleeding. Although gene targeting has recapitulated many of these diseases in mice, these murine homologues are limited as translational models by their small size and brief life span as well as the fact that mutations induced by gene targeting do not always faithfully reflect the clinical manifestations of such… CONTINUE READING