Literature DB >> 24234586

Radiological and clinical characterization of the lysosomal storage disorders: non-lipid disorders.

E I Parker, M Xing, A Moreno-De-Luca, E Harmouche, M R Terk.   

Abstract

Lysosomal storage diseases (LSDs) are a large group of genetic metabolic disorders that result in the accumulation of abnormal material, such as mucopolysaccharides, glycoproteins, amino acids and lipids, within cells. Since many LSDs manifest during infancy or early childhood, with potentially devastating consequences if left untreated, timely identification is imperative to prevent irreversible damage and early death. In this review, the key imaging features of the non-lipid or extralipid LSDs are examined and correlated with salient clinical manifestations and genetic information. Disorders are stratified based on the type of excess material causing tissue or organ dysfunction, with descriptions of the mucopolysaccharidoses, mucolipidoses, alpha-mannosidosis, glycogen storage disorder II and cystinosis. In addition, similarities and differences in radiological findings between each of these LSDs are highlighted to facilitate further recognition. Given the rare and extensive nature of the LSDs, mastery of their multiple clinical and radiological traits may seem challenging. However, an understanding of the distinguishing imaging characteristics of LSDs and their clinical correlates may allow radiologists to play a key role in the early diagnosis of these progressive and potentially fatal disorders.

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Year:  2013        PMID: 24234586      PMCID: PMC3898971          DOI: 10.1259/bjr.20130467

Source DB:  PubMed          Journal:  Br J Radiol        ISSN: 0007-1285            Impact factor:   3.039


  44 in total

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Authors:  Jason S Weisstein; Eliana Delgado; Lynne S Steinbach; Kim Hart; Seymour Packman
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Review 2.  Lysosomal storage disorders: the need for better pediatric recognition and comprehensive care.

Authors:  William R Wilcox
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3.  Brain MRI findings in patients with mucopolysaccharidosis types I and II and mild clinical presentation.

Authors:  M Gisele Matheus; Mauricio Castillo; J Keith Smith; Diane Armao; Diane Towle; Joseph Muenzer
Journal:  Neuroradiology       Date:  2004-06-17       Impact factor: 2.804

4.  The mucopolysaccharidoses and related conditions.

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Journal:  Semin Roentgenol       Date:  1986-10       Impact factor: 0.800

5.  Brain development in infantile-onset Pompe disease treated by enzyme replacement therapy.

Authors:  Yin-Hsiu Chien; Ni-Chung Lee; Shinn-Forng Peng; Wuh-Liang Hwu
Journal:  Pediatr Res       Date:  2006-07-20       Impact factor: 3.756

Review 6.  The clinical presentation of lysosomal storage disorders.

Authors:  James E Wraith
Journal:  Acta Neurol Taiwan       Date:  2004-09

7.  Morquio syndrome: diagnosis in an adult.

Authors:  Catherine Prat; Olivia Lemaire; Johan Bret; Laurent Zabraniecki; Bernard Fournié
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Review 8.  The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature.

Authors:  Hannerieke M P van den Hout; Wim Hop; Otto P van Diggelen; Jan A M Smeitink; G Peter A Smit; Bwee-Tien T Poll-The; Henk D Bakker; M Christa B Loonen; Johannis B C de Klerk; Arnold J J Reuser; Ans T van der Ploeg
Journal:  Pediatrics       Date:  2003-08       Impact factor: 7.124

9.  Mucopolysaccharidosis type VII as a cause of recurrent non-immune hydrops fetalis.

Authors:  Yvonne Cheng; Marion S Verp; Terri Knutel; Judith U Hibbard
Journal:  J Perinat Med       Date:  2003       Impact factor: 1.901

10.  Magnetic resonance imaging in the diagnosis of the cranio-cervical manifestations of the mucopolysaccharidoses.

Authors:  M V Kulkarni; J C Williams; J W Yeakley; J L Andrews; C B McArdle; P A Narayana; R R Howell; A J Jonas
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  4 in total

1.  Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

Authors:  Christian Hinderer; Peter Bell; Brittney L Gurda; Qiang Wang; Jean-Pierre Louboutin; Yanqing Zhu; Jessica Bagel; Patricia O'Donnell; Tracey Sikora; Therese Ruane; Ping Wang; Mark E Haskins; James M Wilson
Journal:  Mol Ther       Date:  2014-07-16       Impact factor: 11.454

2.  Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.

Authors:  Christian Hinderer; Peter Bell; Brittney L Gurda; Qiang Wang; Jean-Pierre Louboutin; Yanqing Zhu; Jessica Bagel; Patricia O'Donnell; Tracey Sikora; Therese Ruane; Ping Wang; Mark E Haskins; James M Wilson
Journal:  Proc Natl Acad Sci U S A       Date:  2014-09-29       Impact factor: 11.205

3.  Skeletal and Brain Abnormalities in Fucosidosis, a Rare Lysosomal Storage Disorder.

Authors:  Camille Malatt; Jeffrey L Koning; John Naheedy
Journal:  J Radiol Case Rep       Date:  2015-05-31

4.  Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler.

Authors:  Xiu Jin; Jing Su; Qinyu Zhao; Ruiting Li; Jianlu Xiao; Xiaomei Zhong; Li Song; Yi Liu; Kaiqin She; Hongxin Deng; Yuquan Wei; Yang Yang
Journal:  Mol Ther Methods Clin Dev       Date:  2022-04-19       Impact factor: 5.849

  4 in total

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