Bone, joint and tooth development in mucopolysaccharidoses: relevance to therapeutic options

Esmee Oussoren; M.M.M.G. Brands; George J. G. Ruijter; Ans T. van der Ploeg; Arnold J. J. Reuser

Bone, joint and tooth development in mucopolysaccharidoses: relevance to therapeutic options

Esmee Oussoren, M.M.M.G. Brands, George J. G. Ruijter, Ans T. van der Ploeg, Arnold J. J. Reuser

Research output: Contribution to journal › Article › Academic › peer-review

Abstract

The mucopolysaccharidoses (MPS) are prominent among the lysosomal storage diseases. The intra-lysosomal accumulation of glycosaminoglycans (GAGs) in this group of diseases, which are caused by several different enzyme deficiencies, induces a cascade of responses that affect cellular functions and maintenance of the extra-cellular matrix. Against the background of normal tissue-specific processes, this review summarizes and discusses the histological and biochemical abnormalities reported in the bones, joints, teeth and extracellular matrix of MPS patients and animal models. With an eye to the possibilities and limitations of reversing the pathological changes in the various tissues, we address therapeutic challenges, and present a model in which the cascade of pathologic events is depicted in terms of primary and secondary events.

Original language	English
Pages (from-to)	1542-56
Journal	BIOCHIMICA ET BIOPHYSICA ACTA
Publication status	Published - Nov 2011

Cite this

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title = "Bone, joint and tooth development in mucopolysaccharidoses: relevance to therapeutic options",

abstract = "The mucopolysaccharidoses (MPS) are prominent among the lysosomal storage diseases. The intra-lysosomal accumulation of glycosaminoglycans (GAGs) in this group of diseases, which are caused by several different enzyme deficiencies, induces a cascade of responses that affect cellular functions and maintenance of the extra-cellular matrix. Against the background of normal tissue-specific processes, this review summarizes and discusses the histological and biochemical abnormalities reported in the bones, joints, teeth and extracellular matrix of MPS patients and animal models. With an eye to the possibilities and limitations of reversing the pathological changes in the various tissues, we address therapeutic challenges, and present a model in which the cascade of pathologic events is depicted in terms of primary and secondary events.",

author = "Esmee Oussoren and M.M.M.G. Brands and Ruijter, {George J. G.} and {van der Ploeg}, {Ans T.} and Reuser, {Arnold J. J.}",

year = "2011",

month = nov,

language = "English",

pages = "1542--56",

journal = "BIOCHIMICA ET BIOPHYSICA ACTA",

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T1 - Bone, joint and tooth development in mucopolysaccharidoses: relevance to therapeutic options

AU - Oussoren, Esmee

AU - Brands, M.M.M.G.

AU - Ruijter, George J. G.

AU - van der Ploeg, Ans T.

AU - Reuser, Arnold J. J.

PY - 2011/11

Y1 - 2011/11

N2 - The mucopolysaccharidoses (MPS) are prominent among the lysosomal storage diseases. The intra-lysosomal accumulation of glycosaminoglycans (GAGs) in this group of diseases, which are caused by several different enzyme deficiencies, induces a cascade of responses that affect cellular functions and maintenance of the extra-cellular matrix. Against the background of normal tissue-specific processes, this review summarizes and discusses the histological and biochemical abnormalities reported in the bones, joints, teeth and extracellular matrix of MPS patients and animal models. With an eye to the possibilities and limitations of reversing the pathological changes in the various tissues, we address therapeutic challenges, and present a model in which the cascade of pathologic events is depicted in terms of primary and secondary events.

AB - The mucopolysaccharidoses (MPS) are prominent among the lysosomal storage diseases. The intra-lysosomal accumulation of glycosaminoglycans (GAGs) in this group of diseases, which are caused by several different enzyme deficiencies, induces a cascade of responses that affect cellular functions and maintenance of the extra-cellular matrix. Against the background of normal tissue-specific processes, this review summarizes and discusses the histological and biochemical abnormalities reported in the bones, joints, teeth and extracellular matrix of MPS patients and animal models. With an eye to the possibilities and limitations of reversing the pathological changes in the various tissues, we address therapeutic challenges, and present a model in which the cascade of pathologic events is depicted in terms of primary and secondary events.

M3 - Article

SN - 0006-3002

SP - 1542

EP - 1556

JO - BIOCHIMICA ET BIOPHYSICA ACTA

JF - BIOCHIMICA ET BIOPHYSICA ACTA

ER -