Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice Article Swipe
Mika Aoyagi-Scharber
,
Danielle Crippen-Harmon
,
Roger Lawrence
,
Jon Vincelette
,
Gouri Yogalingam
,
Heather Prill
,
Bryan K. Yip
,
Brian Baridon
,
Catherine Vitelli
,
Amanda Lee
,
Olivia Gorostiza
,
Evan G. Adintori
,
Wesley Minto
,
Jeremy L. Van Vleet
,
Bridget Yates
,
Sara Rigney
,
Terri Christianson
,
Pascale M.N. Tiger
,
Melanie J. Lo
,
John Holtzinger
,
Paul Fitzpatrick
,
Jonathan H. LeBowitz
,
Sherry Bullens
,
Brett E. Crawford
,
Stuart Bunting
·
YOU?
·
· 2017
· Open Access
·
· DOI: https://doi.org/10.1016/j.omtm.2017.05.009
YOU?
·
· 2017
· Open Access
·
· DOI: https://doi.org/10.1016/j.omtm.2017.05.009
Sanfilippo syndrome type B (mucopolysaccharidosis IIIB), caused by inherited deficiency of α-N-acetylglucosaminidase (NAGLU), required for lysosomal degradation of heparan sulfate (HS), is a pediatric neurodegenerative disorder with no approved treatment. Intracerebroventricular (ICV) delivery of a modified recombinant NAGLU, consisting of human NAGLU fused with insulin-like growth factor 2 (IGF2) for enhanced lysosomal targeting, was previously shown to result in marked enzyme uptake and clearance of HS storage in the Naglu-/- mouse brain. To further evaluate regional, cell type-specific, and dose-dependent biodistribution of NAGLU-IGF2 (BMN 250) and its effects on biochemical and histological pathology, Naglu-/- mice were treated with 1-100 μg ICV doses (four times over 2 weeks). 1 day after the last dose, BMN 250 (100 μg doses) resulted in above-normal NAGLU activity levels, broad biodistribution, and uptake in all cell types, with NAGLU predominantly localized to neurons in the Naglu-/- mouse brain. This led to complete clearance of disease-specific HS and reduction of secondary lysosomal defects and neuropathology across various brain regions lasting for at least 28 days after the last dose. The substantial brain uptake of NAGLU attainable by this highest ICV dosage was required for nearly complete attenuation of disease-driven storage accumulations and neuropathology throughout the Naglu-/- mouse brain.
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- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.1016/j.omtm.2017.05.009
- http://www.cell.com/article/S2329050117300748/pdf
- OA Status
- gold
- Cited By
- 37
- References
- 50
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W2621462985
All OpenAlex metadata
Raw OpenAlex JSON
- OpenAlex ID
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https://openalex.org/W2621462985Canonical identifier for this work in OpenAlex
- DOI
-
https://doi.org/10.1016/j.omtm.2017.05.009Digital Object Identifier
- Title
-
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B MiceWork title
- Type
-
articleOpenAlex work type
- Language
-
enPrimary language
- Publication year
-
2017Year of publication
- Publication date
-
2017-06-06Full publication date if available
- Authors
-
Mika Aoyagi-Scharber, Danielle Crippen-Harmon, Roger Lawrence, Jon Vincelette, Gouri Yogalingam, Heather Prill, Bryan K. Yip, Brian Baridon, Catherine Vitelli, Amanda Lee, Olivia Gorostiza, Evan G. Adintori, Wesley Minto, Jeremy L. Van Vleet, Bridget Yates, Sara Rigney, Terri Christianson, Pascale M.N. Tiger, Melanie J. Lo, John Holtzinger, Paul Fitzpatrick, Jonathan H. LeBowitz, Sherry Bullens, Brett E. Crawford, Stuart BuntingList of authors in order
- Landing page
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https://doi.org/10.1016/j.omtm.2017.05.009Publisher landing page
- PDF URL
-
https://www.cell.com/article/S2329050117300748/pdfDirect link to full text PDF
- Open access
-
YesWhether a free full text is available
- OA status
-
goldOpen access status per OpenAlex
- OA URL
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https://www.cell.com/article/S2329050117300748/pdfDirect OA link when available
- Concepts
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Heparan sulfate, Attenuation, Pathology, Sulfate, Medicine, Endocrinology, Neuroscience, Chemistry, Internal medicine, Biology, Heparin, Physics, Organic chemistry, OpticsTop concepts (fields/topics) attached by OpenAlex
- Cited by
-
37Total citation count in OpenAlex
- Citations by year (recent)
-
2025: 1, 2023: 2, 2022: 5, 2021: 9, 2020: 7Per-year citation counts (last 5 years)
- References (count)
-
50Number of works referenced by this work
- Related works (count)
-
10Other works algorithmically related by OpenAlex
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