Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice.

@article{Herati2008ImprovedRV,
  title={Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice.},
  author={Ramin Sedaghat Herati and Xiucui Ma and Mindy E Tittiger and Kevin K. Ohlemiller and Attila Kov{\'a}cs and Katherine Parker Ponder},
  journal={The journal of gene medicine},
  year={2008},
  volume={10 9},
  pages={972-82}
}
BACKGROUND Mucopolysaccharidosis I (MPS I) is a lysosomal storage disease due to alpha-L-iduronidase (IDUA) deficiency that results in the accumulation of glycosaminoglycans (GAG). Gene therapy can reduce most clinical manifestations, but mice that receive transfer as adults lose expression unless they receive immunosuppression. Increasing liver specificity of transgene expression has reduced immune responses to other genes. METHODS A gamma retroviral vector was generated with a liver… CONTINUE READING

Citations

Publications citing this paper.
Showing 1-10 of 14 extracted citations

References

Publications referenced by this paper.
Showing 1-10 of 42 references

Expression in blood cells may contribute to biochemical and pathological improvements after Copyright 

B Wang, TM O’Malley, L Xu
J Gene Med • 2008
View 1 Excerpt

Expression in blood cells may contribute to biochemical and pathological improvements after Copyright  2008 John Wiley & Sons , Ltd

B Wang, TM O’Malley, L Xu
J Gene Med • 2008

Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy.

Molecular therapy : the journal of the American Society of Gene Therapy • 2007
View 1 Excerpt

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

Molecular therapy : the journal of the American Society of Gene Therapy • 2007
View 10 Excerpts

Similar Papers

Loading similar papers…