Jonathan H LeBowitz Selected Research

alpha-N-acetyl-D-glucosaminidase (alpha-N-acetylglucosaminidase)

1/2020	Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.
9/2019	Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B.
1/2019	Assessment of predicted enzymatic activity of α-N-acetylglucosaminidase variants of unknown significance for CAGI 2016.
1/2019	BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis.
9/2017	Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.
10/2014	Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.

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Jonathan H LeBowitz Research Topics

Disease

5	Mucopolysaccharidosis III (Sanfilippo Syndrome) 09/2019 - 10/2014
4	Lysosomal Storage Diseases (Lysosomal Storage Disease) 01/2020 - 03/2004
3	GM1 Gangliosidosis (Gangliosidosis GM1) 01/2020 - 12/2019
2	Neuraminidase 1 deficiency 11/2020 - 01/2020
2	Mucopolysaccharidoses 09/2019 - 09/2017
2	Neurodegenerative Diseases (Neurodegenerative Disease) 01/2019 - 09/2017
1	Mucolipidoses (Sialidosis) 01/2020
1	Virus Diseases (Viral Diseases) 01/2020
1	Rare Diseases (Rare Disease) 01/2020
1	Inborn Genetic Diseases (Disease, Hereditary) 12/2019
1	Stargardt Disease 10/2017
1	Macular Degeneration (Age-Related Maculopathy) 10/2017
1	Glycogen Storage Disease Type II (Pompe's Disease) 01/2013
1	Mucopolysaccharidosis VII (Sly Syndrome) 03/2004

Drug/Important Bio-Agent (IBA)

6	alpha-N-acetyl-D-glucosaminidase (alpha-N-acetylglucosaminidase)IBA 01/2020 - 10/2014
5	EnzymesIBA 01/2020 - 03/2004
3	BMN 250IBA 01/2020 - 01/2019
3	GalactosidasesIBA 01/2020 - 12/2019
3	Acetylglucosaminidase (NAGase)IBA 09/2019 - 10/2014
3	Heparitin Sulfate (Heparan Sulfate)IBA 01/2019 - 10/2014
2	Galactose (Galactopyranose)FDA LinkGeneric 01/2020 - 12/2019
2	Peptides (Polypeptides)IBA 01/2013 - 03/2004
1	Pharmaceutical PreparationsIBA 01/2020
1	5'-deoxy-5'-phosphonomethyladenosine phosphateIBA 01/2020
1	Recombinant ProteinsIBA 01/2020
1	HydrolasesIBA 12/2019
1	GangliosidesIBA 12/2019
1	GlycolipidsIBA 12/2019
1	GlycoconjugatesIBA 12/2019
1	Biological ProductsIBA 12/2019
1	Polysaccharides (Glycans)IBA 12/2019
1	Insulin-Like PeptidesIBA 09/2019
1	Recombinant Fusion ProteinsIBA 09/2019
1	LipofuscinIBA 10/2017
1	Retinaldehyde (Retinal)IBA 10/2017
1	A2-E (N-retinylidene-N-retinylethanolamine)IBA 10/2017
1	Proteins (Proteins, Gene)FDA Link 10/2014
1	GlucosidasesIBA 01/2013
1	AcidsIBA 01/2013

Therapy/Procedure

8	Enzyme Replacement Therapy 11/2020 - 03/2004
1	Therapeutics 01/2020