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1 features of mucopolysaccharidosis type III (Sanfilippo syndrome).
2 ad to the lysosomal storage disease known as Sanfilippo syndrome.
4 Mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo syndrome B) is an autosomal recessive lysosom
8 common neuropathological findings with other Sanfilippo syndrome models and patients, but they can be
10 ological potential of these glycomimetics in Sanfilippo syndrome, paving the way for the development
11 The first animal disease homolog of human Sanfilippo syndrome type A has been recently indentified
12 e potential development of a canine model of Sanfilippo syndrome type A to evaluate gene therapy for
21 from nine fibroblast cell lines derived from Sanfilippo syndrome type B patients, 10 additional mutat
24 ciently similar to that of patients with the Sanfilippo syndrome type B to make these mice a good mod
25 Mucopolysaccharidosis type IIIB (MPS IIIB, Sanfilippo syndrome type B) is a lysosomal storage disea
27 ministering hUCB cells into a mouse model of Sanfilippo Syndrome type B, the advantages probably a re
29 ns were identified in cells of patients with Sanfilippo syndrome type B: 503del10, R297X, R626X, R643