Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges.
about
Less Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersIntracellular Delivery of Proteins with Cell-Penetrating Peptides for Therapeutic Uses in Human DiseaseImpairment of homeostasis in lysosomal storage disordersEmerging novel concept of chaperone therapies for protein misfolding diseasesLysosomal storage diseases and the heat shock response: convergences and therapeutic opportunitiesOne goal, different strategies--molecular and cellular approaches for the treatment of inherited skin fragility disordersNovel treatment options for lysosomal acid lipase deficiency: critical appraisal of sebelipase alfaGlycan analysis of therapeutic glycoproteinsKey factors influencing ADME properties of therapeutic proteins: A need for ADME characterization in drug discovery and developmentLysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular ToxicityA Novel Functional Missense Mutation p.T219A in Type 1 Gaucher's Disease.Effective intravenous therapy for neurodegenerative disease with a therapeutic enzyme and a peptide that mediates delivery to the brainSimilar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage diseaseVaccination via Chloroplast Genetics: Affordable Protein Drugs for the Prevention and Treatment of Inherited or Infectious Human DiseasesUnlocking a caged lysosomal protein from a polymeric nanogel with a pH trigger.Candidate molecules for chemical chaperone therapy of GM1-gangliosidosis.Carboxyl-terminal truncations alter the activity of the human α-galactosidase APharmacological chaperone therapy for lysosomal storage diseases.Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.Fully human monoclonal antibody inhibitors of the neonatal fc receptor reduce circulating IgG in non-human primates.Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome).Progress in the Development of Small Molecule Therapeutics for the Treatment of Neuronal Ceroid Lipofuscinoses (NCLs).AMP-activated Protein Kinase Suppresses Biosynthesis of Glucosylceramide by Reducing Intracellular Sugar Nucleotides.Phage display as a tool to discover blood-brain barrier (BBB)-shuttle peptides: panning against a human BBB cellular model.Stereodivergent synthesis of right- and left-handed iminoxylitol heterodimers and monomers. Study of their impact on β-glucocerebrosidase activity.Epidemiological, clinical and biochemical characterization of the p.(Ala359Asp) SMPD1 variant causing Niemann-Pick disease type B.Pharmacologic manipulation of lysosomal enzyme transport across the blood-brain barrier.Chitosan-Alginate Microcapsules Provide Gastric Protection and Intestinal Release of ICAM-1-Targeting Nanocarriers, Enabling GI Targeting In Vivo.Characterization of anti-CD20 monoclonal antibody produced by transgenic silkworms (Bombyx mori).The use of lectin microarray for assessing glycosylation of therapeutic proteins.Chaperone-mediated gene therapy with recombinant AAV-PPCA in a new mouse model of type I sialidosisReduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry DiseaseDetermination of 7-ketocholesterol in plasma by LC-MS for rapid diagnosis of acid SMase-deficient Niemann-Pick disease.Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Disease models for the development of therapies for lysosomal storage diseases.A Hitchhiker's guide to the blood-brain barrier: in trans delivery of a therapeutic enzyme.Enzyme replacement therapy for Farber disease: Proof-of-concept studies in cells and mice.Development of pharmacological strategies for mitochondrial disorders.Lysosomal storage diseases--the horizon expands.Pharmaceutical Chaperones and Proteostasis Regulators in the Therapy of Lysosomal Storage Disorders: Current Perspective and Future Promises.
P2860
Q26744230-5D6BED50-5268-40D5-9659-68F4311714D9Q26767093-D6E6F903-B6EA-43A0-ABAC-52EE32607B1CQ26864999-B4DF0615-D7C9-4392-88FD-59517B18AD9FQ27002907-3CC93DCE-3DAE-4652-A038-4141604DDF5DQ27004531-DE6B43D9-522C-43F5-842E-AD57DC1343B1Q27026711-A1294F23-4D4B-446E-9EB5-EEB944F52CE9Q28076525-B58E9A90-D2D5-4F3A-891C-0E0F02BFD9F6Q28082842-86351C8F-A627-4F88-B742-F652637BF3A2Q28082880-19AC4401-D873-494E-9668-36244D631135Q28555118-967723D7-AE71-46D0-B0C3-F6E6F5BA3F6BQ30387151-B7AE1465-6639-41FC-8F8F-9729B0069F73Q30573522-A473172F-6A11-4ABA-8DB1-9B2CA77AF220Q30583745-3A0199AF-487F-40D5-80EC-FC371F74BE7EQ33870122-7E496789-A7DD-4096-8326-554D9815FEFDQ34496997-B04137F5-E74C-429F-869A-1FE3588677C0Q34985010-4616092A-F79E-4EE1-BCE2-871E513B3A4AQ35125851-334E4447-FA60-4BE3-9D8F-94FBF1A2DD81Q35214286-F1CEAA88-8BA3-44F6-995F-99C0CAF6AF75Q35319254-F46EAA7B-2C71-42A0-9740-753F83CD56F3Q35531873-72087EB7-BC71-4739-93BE-35A32FBCD99DQ35559229-DC250076-8887-4CF8-806C-60176A68DC25Q35840603-5074A624-F45A-4319-8B4C-74902502DF39Q35859585-9909ED8F-9657-47ED-BFB4-67A007F47029Q36093633-69D855D2-705B-4283-B86D-A85CE7BA90FAQ36344345-784B46D4-2532-4A3C-8359-0608A4EB3113Q36475065-95398EF9-9EE5-4A6E-B738-521D6195FF7EQ36692494-AC222029-E653-4DAF-9CE4-A8ADECA072ECQ37051502-BE902F00-46FC-4E2C-969E-51D685EAC730Q37138255-BF667C14-9A79-4B71-81A5-596483BB31EEQ37138938-9DDB0271-4D5C-4710-9E6D-BEAEDD35AAC3Q37224436-369109B5-B35A-44A1-A140-91714982A7A4Q37329223-7F1AE139-6A28-403F-BB73-DC50DB560733Q37457283-0FCE6D84-F8CD-454D-B7A6-6140632CADB8Q37507816-A7BF793D-38C7-4E81-BF98-868CF91CA1ABQ37619231-D489E66B-E59F-44A9-98A7-8C363BFE7F95Q37620823-F93AAC0D-8E57-484E-B26D-4A48C64A04BFQ37682583-5D13CEA0-64AF-4416-AA2C-74706B6774B2Q37686237-D78BD9E1-C0BF-4B1E-A31B-E5D5712775A6Q38128478-E1A4B103-0365-4F83-9E93-590E5F5DDF03Q38652950-1CE9FB08-1673-415B-9D2C-AF6F497DA939
P2860
Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges.
description
article científic
@ca
article scientifique
@fr
articol științific
@ro
articolo scientifico
@it
artigo científico
@gl
artigo científico
@pt
artigo científico
@pt-br
artikel ilmiah
@id
artikull shkencor
@sq
artículo científico
@es
name
Enzyme replacement therapy for ...... ence and remaining challenges.
@en
type
label
Enzyme replacement therapy for ...... ence and remaining challenges.
@en
prefLabel
Enzyme replacement therapy for ...... ence and remaining challenges.
@en
P1476
Enzyme replacement therapy for ...... ence and remaining challenges.
@en
P2093
E H Schuchman
R J Desnick
P304
P356
10.1146/ANNUREV-GENOM-090711-163739
P577
2012-01-01T00:00:00Z