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: 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

المؤلفون: Shih-hsin Kan, Zhi Chen, Stuart Bunting, Brett E. Crawford, Wesley Wong, Anil Bagri, Evan G. Adintori, John Holtzinger, Melanie J. Lo, Elizabeth F. Neufeld, Danielle Crippen-Harmon, Paul A. Fitzpatrick, Jillian R. Brown, Josh C. Woloszynek, Pascale M.N. Tiger, Kristen N. Vondrak, Diana S. Cheung, Kazuhiro Ohmi, Jon Vincelette, Sherry Bullens, Jonathan H. LeBowitz, Chuck Hague, Steven Q. Le, Roger Lawrence, Terri Christianson, Bryan K. Yip, Daniel J. Wendt, Katherine A. Webster, Mika Aoyagi-Scharber, Patricia I. Dickson

المصدر: Proceedings of the National Academy of Sciences of the United States of America, vol 111, iss 41

مصطلحات موضوعية: Aging, Mucopolysaccharidoses (MPS), Neurodegenerative, Alzheimer's Disease, chemistry.chemical_compound, Mice, Mucopolysaccharidosis III, Drug Delivery Systems, Cricetinae, Lysosomal storage disease, Cells, Cultured, Sanfilippo syndrome, Neurons, Multidisciplinary, Cultured, LAMP1, Brain, Heparan sulfate, Enzyme replacement therapy, Biological Sciences, Endocytosis, beta-N-Acetylhexosaminidases, Liver, Neurological, Biotechnology, Protein Binding, Protein subunit, Cells, Intraventricular, Recombinant Fusion Proteins, CHO Cells, Biology, Injections, Rare Diseases, Cricetulus, Insulin-Like Growth Factor II, Acetylglucosaminidase, medicine, Acquired Cognitive Impairment, Animals, Humans, Metabolic and endocrine, Injections, Intraventricular, Neurosciences, Alzheimer's Disease including Alzheimer's Disease Related Dementias (AD/ADRD), Lysosome-Associated Membrane Glycoproteins, Fibroblasts, medicine.disease, Fusion protein, Molecular biology, Brain Disorders, Orphan Drug, chemistry, Dementia, Heparitin Sulfate, Biomarkers

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f6e9cef317d3d7e5f182f493e8a4dd4cTest
https://europepmc.org/articles/PMC4205671Test/

المؤلفون: Jillian R. Brown, Danielle Crippen-Harmon, Stuart Bunting, Paul A. Fitzpatrick, Mika Aoyagi-Scharber, Anil Bagri, Sherry Bullens, John Holtzinger, Bryan K. Yip, Jon Vincelette, Heather Prill, Roger Lawrence, Evan G. Adintori, Wesley C. Minto, Eric Chen, Jeremy Van Vleet, Pascale M.N. Tiger, Brian Baridon, Catherine Vitelli, Brett E. Crawford, Terri Christianson, Melanie J. Lo, Jonathan H. LeBowitz, Katherine A. Webster

المصدر: Molecular Genetics and Metabolism. 117:S21

مصطلحات موضوعية: Normalization (statistics), medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Mucopolysaccharidosis, Dose dependence, Enzyme replacement therapy, medicine.disease, Biochemistry, Endocrinology, Internal medicine, Genetics, medicine, business, Molecular Biology, Pathological, Sanfilippo syndrome

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::5b8b57352c2844156870701864495a0fTest
https://doi.org/10.1016/j.ymgme.2015.12.180Test