Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
about
Molecular basis of reduced glucosylceramidase activity in the most common Gaucher disease mutant, N370SProduction of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell systemNovel patient cell-based HTS assay for identification of small molecules for a lysosomal storage diseaseBilateral symmetrical cortical osteolytic lesions in two patients with Gaucher disease.Glycosylation of therapeutic proteins: an effective strategy to optimize efficacyA breach in the blood-brain barrier.Type 2 Gaucher disease: phenotypic variation and genotypic heterogeneity.Gaucher disease: pathological mechanisms and modern management.A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B.Azasugar inhibitors as pharmacological chaperones for Krabbe diseaseActivation of p38 Mitogen-Activated Protein Kinase in Gaucher's DiseaseRecent advances towards development and commercialization of plant cell culture processes for the synthesis of biomoleculesThe cell biology of lysosomal storage disorders.Direct multiplex assay of lysosomal enzymes in dried blood spots for newborn screening.Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII miceA survey of the pain experienced by males and females with Fabry diseaseMacrophage differentiation and function in health and disease.Structural glycobiology: a game of snakes and ladders.Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier.Characterization and application of a disease-cell model for a neurodegenerative lysosomal disease.Clinical neurogenetics: neuropathic lysosomal storage disorders.Progress and potential of non-inhibitory small molecule chaperones for the treatment of Gaucher disease and its implications for Parkinson disease.Secondary alterations of sphingolipid metabolism in lysosomal storage diseases.Gaucher disease types 1 and 3: Phenotypic characterization of large populations from the ICGG Gaucher Registry.Lysosome-targeted octadecyl-rhodamine B-liposomes enhance lysosomal accumulation of glucocerebrosidase in Gaucher's cells in vitro.Lysosomal delivery of therapeutic enzymes in cell models of Fabry disease.An integrated chip for immunofluorescence and its application to analyze lysosomal storage disorders.Targeting of lysosomes by liposomes modified with octadecyl-rhodamine BGlycosaminoglycans modulate activation, activity, and stability of tripeptidyl-peptidase I in vitro and in vivo.Immunogenicity of glycans on biotherapeutic drugs produced in plant expression systems-The taliglucerase alfa story.Oxidative stress, trace elements, and circulating microparticles in patients with Gaucher disease before and after enzyme replacement therapy.Conditional expression of human acid beta-glucosidase improves the visceral phenotype in a Gaucher disease mouse model.
P2860
Q24621867-471F0844-203F-40E3-98D0-A47CAAAD7659Q27644895-DD4ADEBA-F23D-4C57-9312-9BAEFC02EAE9Q28478533-7F23E88B-6775-4198-AB08-753FE3CB6AB7Q30426973-3A9F6598-3208-4BB9-937E-7BEA7F8E5FFEQ33586660-04E8360A-3F6E-40D8-9456-5F009112740BQ34078637-3BB52918-5684-4879-A859-03B4748CE6AEQ34479337-27C06CF2-4236-4330-9980-5CD2EB58F6D8Q34556549-7BFD69BD-153F-4545-8989-A101E2C27C4FQ34795793-F7B6683F-4A02-416A-9BC4-9759B8853FF9Q35652295-FFC44028-9DCA-43AF-A784-CF0A1C80D43FQ35757092-E5EF8969-624F-4D1F-89C3-B4EE3FB50C6DQ35784729-2E5585BE-9DE3-4CAA-A879-3A11570FB469Q35825859-60EBF5A2-0138-4D8E-B89B-8C0D81226D4EQ36193022-AB488C0F-0F14-4790-8CA9-032A115A601BQ36853823-A2FE972B-961D-41C8-8F9C-1881071FB90FQ36883412-C153C19B-6D27-4893-9DE3-E6B4BA35E763Q37077690-50321757-F3F9-452C-BA6C-CC1755B5AA5FQ37129248-DA06753D-EADF-4A98-BD0D-B04CE4083AA3Q37494188-79CD93E5-6A0C-4B91-A63F-80A4AD8E62F1Q37624819-F1F6D7D5-D815-4E6F-9EE8-F2EE234F26E0Q37705073-9062CF0C-05B4-4452-8A3A-29C9C01E0830Q37738863-7D9CBE30-85B2-495A-B3C6-0E4257F3F1FEQ37826106-5B75550E-80E3-4B09-9F0E-6D652B227A35Q38533848-4A5F3893-99D6-4A29-9184-3BCC1FF06713Q39235292-D4EF0746-7115-4A2C-A272-E2ACA4350641Q39373577-2ADF16B7-7132-4885-9F8E-B96E47B2F230Q39436051-3F095508-853B-4A43-9638-4AEAD18B78FEQ42759018-DAADA8D8-D241-44E8-9C3B-4C144EA7D7F0Q45176585-8EA8F0C5-A5FB-498E-9FEA-A61B823FEC38Q45437801-288E4241-C3E5-45D1-9216-190216D718FFQ46241928-78566622-4354-429B-8C3F-3E52B48C1E4FQ48464562-DAAEAAEF-15CE-4941-A154-AE1EB9997BDF
P2860
Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
description
2003 nî lūn-bûn
@nan
2003年の論文
@ja
2003年論文
@yue
2003年論文
@zh-hant
2003年論文
@zh-hk
2003年論文
@zh-mo
2003年論文
@zh-tw
2003年论文
@wuu
2003年论文
@zh
2003年论文
@zh-cn
name
Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
@ast
Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
@en
type
label
Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
@ast
Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
@en
prefLabel
Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
@ast
Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
@en
P1476
Enzyme therapy for lysosomal storage disease: principles, practice, and prospects.
@en
P2093
Gregory A Grabowski
P304
P356
10.1146/ANNUREV.GENOM.4.070802.110415
P577
2003-01-01T00:00:00Z