8 results on '"LeBowitz, Jonathan H."'
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2. BMN 701-mediated receptor redistribution is responsible for increased uptake.
3. Histologic characterization of the progression of central nervous system pathology in the mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo syndrome type B) mouse model and bio-distribution and efficacy of the intracerebroventricular enzyme replacement therapy, BMN 250
4. Immunohistochemical analysis of mannose 6-phosphate/insulin-like growth factor 2 receptor in murine wild-type and mucopolysaccharidosis IIIB mutant central nervous system vasculature and implications for trans-blood brain barrier (BBB) transport.
5. Evaluation of myeloperoxidase as a targeted enzymatic approach for the elimination of retinal A2E in Stargardt disease.
6. Glycosylation independent lysosomal targeting of alpha-n-acetylglucosaminidase confers highly efficient enzyme uptake into critical cellular targets of disease pathogenesis in mucopolysaccharidosis type IIIB.
7. Time- and dose-dependent normalization of pathological lysosomal storage and biochemistry in the mucopolysaccharidosis ΙΙΙΒ (MPS ΙΙΙΒ, Sanfilippo syndrome type Β) mouse model by intracerebroventricular enzyme replacement therapy with ΒΜΝ 250, a ΝAGLU-ΙGF2 fusion pro
8. Engineering of a recombinant NAGLU fusion protein with insulin-like growth factor 2 leads to improved cellular uptake via a glycosylation-independent lysosomal targeting pathway.
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