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