Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.

@article{Cubizolle2014CorrectiveGT,
  title={Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.},
  author={Aur{\'e}lie Cubizolle and Nicolas Serratrice and Nadia Skander and Marie-Anne Colle and Sandy Ibanes and Aur{\'e}lie Gennetier and Neus Bayo-Puxan and Khalil Mazouni and Franck J.D. Mennechet and B{\'e}atrice Joussemet and Yan Chérel and Yaouen Lajat and Charles H. Vite and Florence Bernex and Vasiliki Kalatzis and Mark E. Haskins and Eric J. Kremer},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2014},
  volume={22 4},
  pages={
          762-73
        }
}
Severe deficiency in lysosomal β-glucuronidase (β-glu) enzymatic activity results in mucopolysaccharidosis (MPS) VII, an orphan disease with symptoms often appearing in early childhood. Symptoms are variable, but many patients have multiple organ disorders including neurological defects. At the cellular level, deficiency in β-glu activity leads to abnormal accumulation of glycosaminoglycans (GAGs), and secondary accumulation of GM2 and GM3 gangliosides, which have been linked to… 
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