Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB

Mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo syndrome B; OMIM #252920) is a lethal, pediatric, neuropathic, autosomal recessive, and lysosomal storage disease with no approved therapy. Patients are deficient in the activity of N-acetyl-alpha-glucosaminidase (NAGLU; EC 3.2.150), necessary fo...

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Detalles Bibliográficos
Autores principales: Ellinwood, N. Matthew, Valentine, Bethann N., Hess, Andrew S., Jens, Jackie K., Snella, Elizabeth M., Jamil, Maryam, Hostetter, Shannon J., Jeffery, Nicholas D., Smith, Jodi D., Millman, Suzanne T., Parsons, Rebecca L., Butt, Mark T., Chandra, Sundeep, Egeland, Martin T., Assis, Ana B., Nelvagal, Hemanth R., Cooper, Jonathan D., Nestrasil, Igor, Mueller, Bryon A., Labounek, Rene, Paulson, Amy, Prill, Heather, Liu, Xiao Ying, Zhou, Huiyu, Lawrence, Roger, Crawford, Brett E., Grover, Anita, Cherala, Ganesh, Melton, Andrew C., Cherukuri, Anu, Vuillemenot, Brian R., Wait, Jill C.M., O’Neill, Charles A., Pinkstaff, Jason, Kovalchin, Joseph, Zanelli, Eric, McCullagh, Emma
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The American Society for Pharmacology and Experimental Therapeutics 2022
Materias:
Drug Discovery and Translational Medicine
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9426762/
https://www.ncbi.nlm.nih.gov/pubmed/35717448
http://dx.doi.org/10.1124/jpet.122.001119

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