المؤلفون: 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 C. Minto, Jeremy L. Van Vleet, Bridget Yates, Sara Rigney, Terri M. Christianson, Pascale M.N. Tiger, Melanie J. Lo, John Holtzinger, Paul A. Fitzpatrick, Jonathan H. LeBowitz, Sherry Bullens, Brett E. Crawford, Stuart Bunting

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 6, Iss C, Pp 43-53 (2017)

مصطلحات موضوعية: lysosomal storage disorder, mucopolysaccharidosis IIIB, MPS IIIB, Sanfilippo B, enzyme replacement therapy, intracerebroventricular delivery, glycosaminoglycan, neurodegeneration, mental retardation, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

العلاقة: http://www.sciencedirect.com/science/article/pii/S2329050117300748Test; https://doaj.org/toc/2329-0501Test

الوصول الحر: https://doaj.org/article/391415b4d80b4db49263e27fbcc2640aTest

المؤلفون: Alexander Giaramita, Nathan Wise, Jon Vincelette, Rolando De Angelis, Brett E. Crawford, Sherry Bullens, Nicole Galicia, Melanie J. Lo, Jeremy L. Van Vleet, Sushmita M Roy, Gouri Yogalingam, Amanda R. Luu, Linley Mangini, Alessandra d'Azzo, Vishal Agrawal, Glenn Pacheco, Sylvia Fong, Jonathan H. LeBowitz, Terri Christianson, Hio Wong, Roger Lawrence, Britta Handyside, Harry J Sterling, Stuart Bunting, Chuck Hague, Joseph C Chen

المصدر: The Journal of Biological Chemistry

مصطلحات موضوعية: 0301 basic medicine, safety, medicine.medical_specialty, GM1 gangliosidosis, Biochemistry, enzyme replacement therapy (ERT), 03 medical and health sciences, Mice, Internal medicine, Lysosome, biophysics, Lysosomal storage disease, medicine, Animals, cation-independent mannose-6-phosphate receptor, Enzyme Replacement Therapy, unfolded protein response (UPR), Molecular Biology, Ganglioside, Gangliosidosis, GM1, 030102 biochemistry & molecular biology, Chemistry, Chinese hamster ovary cell, Endoplasmic reticulum, neurodegeneration, toxicity, Enzyme replacement therapy, Cell Biology, medicine.disease, beta-Galactosidase, gene therapy, 030104 developmental biology, medicine.anatomical_structure, Endocrinology, GLB1, lysosomal storage disease, Unfolded protein response, lysosome, endoplasmic reticulum stress

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1842a37d379f3461db62d067305f34f9Test
http://europepmc.org/articles/PMC7521651Test

المؤلفون: Jeremy L. Van Vleet, Mika Aoyagi-Scharber, Bryan K. Yip, Paul A. Fitzpatrick, Terri Christianson, Olivia Gorostiza, Gouri Yogalingam, Danielle Crippen-Harmon, Stuart Bunting, Heather Prill, Pascale M.N. Tiger, Evan G. Adintori, Sherry Bullens, Jonathan H. LeBowitz, Wesley C. Minto, Catherine Vitelli, Bridget Yates, Brian Baridon, Roger Lawrence, Brett E. Crawford, John Holtzinger, Jon Vincelette, Sara Rigney, Amanda Lee, Melanie J. Lo

المصدر: Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 6, Iss C, Pp 43-53 (2017)

مصطلحات موضوعية: 0301 basic medicine, Biodistribution, Pathology, medicine.medical_specialty, lcsh:QH426-470, Mucopolysaccharidosis, medicine.medical_treatment, Neuropathology, mental retardation, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Internal medicine, Genetics, medicine, glycosaminoglycan, lcsh:QH573-671, Molecular Biology, Sanfilippo syndrome, intracerebroventricular delivery, lcsh:Cytology, Growth factor, Neurodegeneration, neurodegeneration, Heparan sulfate, Enzyme replacement therapy, medicine.disease, mucopolysaccharidosis IIIB, lcsh:Genetics, 030104 developmental biology, Endocrinology, chemistry, MPS IIIB, Sanfilippo B, Molecular Medicine, Original Article, lysosomal storage disorder, 030217 neurology & neurosurgery, enzyme replacement therapy

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::be65c6cfa3b3eb67e29a91c8c3b7e7a5Test
https://pubmed.ncbi.nlm.nih.gov/28664165Test