Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle-Reference-Cited by-同舟云学术

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Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

Published:2011-06 Issue:2 Volume:103 Page:107-112
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Koeberl Dwight D.,Luo Xiaoyan,Sun Baodong,McVie-Wylie Alison,Dai Jian,Li Songtao,Banugaria Suhrad G.,Chen Y.-T.,Bali Deeksha S.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference34 articles.

1. Glycogen storage disease type II: acid α-glucosidase (acid maltase) deficiency;Hirschhorn,2001

2. Recombinant human acid {alpha}-glucosidase: major clinical benefits in infantile-onset Pompe disease;Kishnani;Neurology,2007

3. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants;Kishnani;Mol. Genet. Metab.,2010

4. Murine acid α-glucosidase: cell-specific mRNA differential expression during development and maturation;Ponce;Am. J. Pathol.,1999

5. Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial;Strothotte;J. Neurol.,2010

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1. Clinical insight meets scientific innovation to develop a next generation ERT for Pompe disease;Molecular Genetics and Metabolism;2024-09

2. Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells;Molecular Therapy - Nucleic Acids;2024-06

3. Small-molecule inhibition of glycogen synthase 1 for the treatment of Pompe disease and other glycogen storage disorders;Science Translational Medicine;2024-01-17

4. Cation-independent mannose 6-phosphate receptor: From roles and functions to targeted therapies;Journal of Controlled Release;2024-01

5. Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice;Journal of Pharmacology and Experimental Therapeutics;2023-09-07

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