Literature DB >> 15119106

Enzyme replacement therapy for lysosomal storage disorders: successful transition from concept to clinical practice.

William S Sly1.   

Abstract

Lysosomal disorders offer a striking example of how basic scientific research has been translated into effective medical intervention. The successful application of enzyme replacement therapy for several lysosomal disorders in animal models and in human disease offers hope for everexpanding abilities to counter the effects of these disorders.

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Year:  2004        PMID： 15119106

Source DB:  PubMed          Journal:  Mo Med        ISSN： 0026-6620


  9 in total

Review 1.  Gene therapy for the neurological manifestations in lysosomal storage disorders.

Authors:  Seng H Cheng
Journal:  J Lipid Res       Date:  2014-03-29       Impact factor: 5.922

Review 2.  Acid alpha-glucosidase deficiency (Pompe disease).

Authors:  Tokiko Fukuda; Ashley Roberts; Paul H Plotz; Nina Raben
Journal:  Curr Neurol Neurosci Rep       Date:  2007-01       Impact factor: 5.081

3.  Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice.

Authors:  Yunxiang Zhu; Xuemei Li; Alison McVie-Wylie; Canwen Jiang; Beth L Thurberg; Nina Raben; Robert J Mattaliano; Seng H Cheng
Journal:  Biochem J       Date:  2005-08-01       Impact factor: 3.857

4.  Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease.

Authors:  Tokiko Fukuda; Meghan Ahearn; Ashley Roberts; Robert J Mattaliano; Kristien Zaal; Evelyn Ralston; Paul H Plotz; Nina Raben
Journal:  Mol Ther       Date:  2006-09-27       Impact factor: 11.454

Review 5.  Enzyme replacement therapy of Fabry disease.

Authors:  Joe T R Clarke; R Mark Iwanochko
Journal:  Mol Neurobiol       Date:  2005-08       Impact factor: 5.590

6.  Involvement of the Toll-like receptor 4 pathway and use of TNF-alpha antagonists for treatment of the mucopolysaccharidoses.

Authors:  Calogera M Simonaro; Yi Ge; Efrat Eliyahu; Xingxuan He; Karl J Jepsen; Edward H Schuchman
Journal:  Proc Natl Acad Sci U S A       Date:  2009-12-14       Impact factor: 11.205

Review 7.  Chemoenzymatic Methods for the Synthesis of Glycoproteins.

Authors:  Chao Li; Lai-Xi Wang
Journal:  Chem Rev       Date:  2018-08-24       Impact factor: 60.622

8.  Chemoenzymatic Synthesis and Receptor Binding of Mannose-6-Phosphate (M6P)-Containing Glycoprotein Ligands Reveal Unusual Structural Requirements for M6P Receptor Recognition.

Authors:  Takahiro Yamaguchi; Mohammed N Amin; Christian Toonstra; Lai-Xi Wang
Journal:  J Am Chem Soc       Date:  2016-08-16       Impact factor: 15.419

9.  Altered lumbar spine structure, biochemistry, and biomechanical properties in a canine model of mucopolysaccharidosis type VII.

Authors:  Lachlan J Smith; John T Martin; Spencer E Szczesny; Katherine P Ponder; Mark E Haskins; Dawn M Elliott
Journal:  J Orthop Res       Date:  2010-05       Impact factor: 3.494
  9 in total

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