Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.

  title={Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.},
  author={Aur{\'e}lie Cubizolle and Nicolas Serratrice and N. Skander and M. Colle and Sandy Ibanes and Aur{\'e}lie Gennetier and Neus Bayo-Puxan and K. Mazouni and Franck J. D. Mennechet and B. Joussemet and Y. Ch{\'e}rel and Y. Lajat and C. Vite and F. Bernex and V. Kalatzis and M. Haskins and E. Kremer},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  volume={22 4},
  • Aurélie Cubizolle, Nicolas Serratrice, +14 authors E. Kremer
  • Published 2014
  • Biology, Medicine
  • Molecular therapy : the journal of the American Society of Gene Therapy
  • 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… CONTINUE READING
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