Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
about
The 1.9 a structure of human alpha-N-acetylgalactosaminidase: The molecular basis of Schindler and Kanzaki diseasesA unique region of RILP distinguishes it from its related proteins in its regulation of lysosomal morphology and interaction with Rab7 and Rab34Protein homeostasis as a therapeutic target for diseases of protein conformationCatalytic mechanism of human alpha-galactosidaseA bacterial glycosidase enables mannose-6-phosphate modification and improved cellular uptake of yeast-produced recombinant human lysosomal enzymesNanoparticles and intracellular applications of surface-enhanced Raman spectroscopyAptamer-Drug ConjugatesLate endosomal cholesterol accumulation leads to impaired intra-endosomal traffickingPartial restoration of mutant enzyme homeostasis in three distinct lysosomal storage disease cell lines by altering calcium homeostasisGDNF-transfected macrophages produce potent neuroprotective effects in Parkinson's disease mouse modelLong-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transferPyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis.Lending a helping hand, screening chemical libraries for compounds that enhance beta-hexosaminidase A activity in GM2 gangliosidosis cellsLysosomal alpha-galactosidase controls the generation of self lipid antigens for natural killer T cellsCell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidosesSebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency.Type 2 Gaucher disease: phenotypic variation and genotypic heterogeneity.Gaucher disease: pathological mechanisms and modern management.Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses.High incidence of later-onset fabry disease revealed by newborn screeningEnzyme replacement therapy for Fabry disease: some answers but more questionsCarboxyl-terminal truncations alter the activity of the human α-galactosidase AExosomes as drug delivery vehicles for Parkinson's disease therapyPreclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis.New technologies for drug delivery across the blood brain barrier.The cell biology of lysosomal storage disorders.A review of gene therapy in canine and feline models of lysosomal storage disorders.Induced Pluripotent Stem Cells for Disease Modeling and Evaluation of Therapeutics for Niemann-Pick Disease Type APharmacological Chaperoning: A Potential Treatment for PMM2-CDG.Fasting activates macroautophagy in neurons of Alzheimer's disease mouse model but is insufficient to degrade amyloid-betaChallenges in design and characterization of ligand-targeted drug delivery systems.Fabry disease: identification of 50 novel alpha-galactosidase A mutations causing the classic phenotype and three-dimensional structural analysis of 29 missense mutations.Comparative binding, endocytosis, and biodistribution of antibodies and antibody-coated carriers for targeted delivery of lysosomal enzymes to ICAM-1 versus transferrin receptorGenetic diseases of sphingolipid metabolism: pathological mechanisms and therapeutic options.Pharmacologic manipulation of lysosomal enzyme transport across the blood-brain barrier.Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII miceAptamer-based endocytosis of a lysosomal enzyme.Defining the pathway for Tat-mediated delivery of beta-glucuronidase in cultured cells and MPS VII mice.Systems pharmacology and genome medicine: a future perspective.A macrophage-nanozyme delivery system for Parkinson's disease.
P2860
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P2860
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
description
2002 nî lūn-bûn
@nan
2002 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2002 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2002年の論文
@ja
2002年論文
@yue
2002年論文
@zh-hant
2002年論文
@zh-hk
2002年論文
@zh-mo
2002年論文
@zh-tw
2002年论文
@wuu
name
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
@ast
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
@en
type
label
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
@ast
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
@en
prefLabel
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
@ast
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
@en
P356
P1476
Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.
@en
P2093
Edward H Schuchman
Robert J Desnick
P2888
P304
P356
10.1038/NRG963
P577
2002-12-01T00:00:00Z
P6179
1052731625