Neurofilament light is a treatment‐responsive biomarker in CLN2 disease

Autor: Russell K. Soon, Temitayo Ajayi, Nancy Pryer, Guoying K. Yu, John Sinclair, Sanjay Chandriani, Andrew Melton, Carley R. Corado, Yuanbin Ru, Adam Harris, Chris B. Russell, David Jacoby, Martin L. Katz

Publikováno v: Annals of Clinical and Translational Neurology
Annals of Clinical and Translational Neurology, Vol 6, Iss 12, Pp 2437-2447 (2019)

Objective Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare, progressive, fatal neurodegenerative pediatric disorder resulting from deficiencies of the lysosomal enzyme tripeptidyl peptidase 1 that are caused by mutations in TPP1. Identi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7b1f747839ea2150bd81acb05cdddfc9
http://europepmc.org/articles/PMC6917340

Antibodies that neutralize cellular uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndrome

Autor: Stephen J. Zoog, Charles A. O'Neill, Troy Tompkins, Yulan Qi, Andrew Melton, Russell K. Soon, Catherine Vitelli, Celeste Decker, Brian Long, Anil Bagri, Becky Schweighardt, Lynne Jesaitis

Publikováno v: Journal of Immunological Methods. 440:41-51

Many enzyme replacement therapies (ERTs) for lysosomal storage disorders use the cell-surface cation-independent mannose-6 phosphate receptor (CI-M6PR) to deliver ERTs to the lysosome. However, neutralizing antibodies (NAb) may interfere with this pr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2eff7029124c48775af6d25488575f2e
https://doi.org/10.1016/j.jim.2016.10.006

Detection of Antibodies That Neutralize the Cellular Uptake of Enzyme Replacement Therapies with a Cell-based Assay

Autor: Stephen J. Zoog, Andrew Melton, Lynne Jesaitis, Gregory W. deHart, Ruby Cheung

Publikováno v: Journal of Visualized Experiments : JoVE

The administration of enzyme replacement therapies (ERTs) and other biologic therapies to patients may elicit an anti-drug immune response. The characterization of these anti-drug antibodies (ADA), especially those that may neutralize the biological

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6fcaea2f9ea9c9abd5ba55aeb7197975
http://europepmc.org/articles/PMC6235114

ICV-administered tralesinidase alfa (BMN 250 NAGLU-IGF2) is well-tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

Autor: Stephen M. Maricich, Maureen Cleary, İlyas Okur, Shuan-Pei Lin, Nicole Muschol, Heather Cahan, Lynn Smith, María L. Couce, Joy Lee, Heidi Peters, Martha Solano Villarreal, Anita Grover, Paul Harmatz, Maria Jose de Castro Lopez, Adam J. Shaywitz, Andrew Melton, Fatih Süheyl Ezgü

Publikováno v: Molecular Genetics and Metabolism. 126:S40

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c00720e2aaed856989011fb45c4dbe0a
https://doi.org/10.1016/j.ymgme.2018.12.083

Growth of pattern-free InN micropyramids by metalorganic chemical vapor deposition

Autor: Ian T. Ferguson, Balakrishnam Jampana, Tahir Zaidi, Muhammad Jamil, Andrew Melton, Tianming Xu, Boon S. Ooi, C.L. Tan

Publikováno v: physica status solidi (a). 207:1895-1899

We report on the growth of unidirectional InN micropyramids by using a simple pattern-free epitaxial technique. These pyramids were grown on a GaN layer deposited on sapphire via metalorganic chemical vapor deposition (MOCVD). The XRD full width at h

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::6d521e6cdf56ae0e88b96101951d9977
https://doi.org/10.1002/pssa.200925462