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Gene therapy of the brain in the dog model of Hurler's syndrome.
Carine C Ciron, Nathalie N Desmaris, Marie-Anne MA Colle, Sylvie S Raoul, Béatrice B Joussemet, Lucie L Vérot, Jérôme J Ausseil, Roseline R Froissart, Françoise F Roux, Yan Y Chérel, Nicolas N Ferry, Yaouen Y Lajat, Bertrand B Schwartz, Marie-Thérèse MT Vanier, Irène I Maire, Marc M Tardieu, Philippe P Moullier and Jean-Michel JM Heard
Ann Neurol 60(2):204-13 (2006)
PMID 16718701
A defect of the lysosomal enzyme alpha-L-iduronidase (IDUA) interrupts the degradation of glycosaminoglycans in mucopolysaccharidosis type I, causing severe neurological manifestations in children with Hurler's syndrome. Delivery of the missing enzyme through stereotactic injection of adeno-associated virus vectors coding for IDUA prevents neuropathology in affected mice. We examined the efficacy and the safety of this approach in enzyme-deficient dogs.
DOI: 10.1002/ana.20870
Version: za2963e q8zaa q8zb0 q8zc5 q8zdd q8zea q8zf9 q8zg5
DOI: 10.1002/ana.20870
Version: za2963e q8zaa q8zb0 q8zc5 q8zdd q8zea q8zf9 q8zg5