Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in pompe mice.
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Targeted approaches to induce immune tolerance for Pompe disease therapyImmune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent.Pompe disease: from pathophysiology to therapy and back againDelivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe DiseaseEnzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Pompe disease: literature review and case seriesA chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy.Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe miceDiagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert groupOral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice.Pompe disease: Shared and unshared features of lysosomal storage disordersProduction of a functional human acid maltase in tobacco seeds: biochemical analysis, uptake by human GSDII cells, and in vivo studies in GAA knockout miceA beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease.Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center studyTranscription factor EB (TFEB) is a new therapeutic target for Pompe diseaseSkeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy.The respiratory neuromuscular system in Pompe disease.Lysosomal storage diseases--the horizon expands.Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel.Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease.Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe diseaseAAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease.Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy.Design of Potent Mannose 6-Phosphate Analogues for the Functionalization of Lysosomal Enzymes To Improve the Treatment of Pompe Disease.Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms.
P2860
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P2860
Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in pompe mice.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
2012年论文
@zh
2012年论文
@zh-cn
name
Glycosylation-independent lyso ...... cogen clearance in pompe mice.
@ast
Glycosylation-independent lyso ...... cogen clearance in pompe mice.
@en
type
label
Glycosylation-independent lyso ...... cogen clearance in pompe mice.
@ast
Glycosylation-independent lyso ...... cogen clearance in pompe mice.
@en
prefLabel
Glycosylation-independent lyso ...... cogen clearance in pompe mice.
@ast
Glycosylation-independent lyso ...... cogen clearance in pompe mice.
@en
P2093
P2860
P356
P1476
Glycosylation-independent lyso ...... cogen clearance in pompe mice.
@en
P2093
Angela Thomm
Barry J Byrne
Jianghong Zhou
John A Maga
Jonathan H LeBowitz
Nancy M Dahms
Ravi Kambampati
Richard N Bohnsack
Sarah Golata
P2860
P304
P356
10.1074/JBC.M112.438663
P407
P577
2012-11-27T00:00:00Z