Literature DB >> 18509743

Clinical characterization of cardiovascular abnormalities associated with feline mucopolysaccharidosis I and VI.

M M Sleeper1, C M Kusiak, F S Shofer, P O'Donnell, C Bryan, K P Ponder, M E Haskins.   

Abstract

OBJECTIVE: The purpose of this study was to define the cardiovascular abnormalities present in young and adult cats affected with the lysosomal storage diseases mucopolysaccharidosis (MPS) I and MPS VI.
METHOD: Eighteen cats affected with MPS I and 10 cats affected with MPS VI were evaluated by physical examination, electrocardiography and echocardiography. Electrocardiography (ECG) was performed on all MPS I and 9 of the MPS VI cats. Twelve unaffected cats underwent complete examinations for comparison purposes.
RESULTS: No cardiovascular abnormalities were noted on physical examination. Measured ECG intervals were normal in affected cats; however, sinus arrhythmia was noted more frequently than in the unaffected cats. Significant echocardiographic abnormalities included aortic valve thickening, regurgitation and aortic root dilation. Significant mitral valve thickening was also noted. The severity of changes increased in older affected cats.
CONCLUSION: As affected animals increased in age, more cardiac abnormalities were found with increasing severity. Significant lesions included the mitral and aortic valves and ascending aorta, but myocardial changes were not recognized. MPS I and MPS VI cats have similar cardiovascular findings to those seen in children and constitute important models for testing new MPS therapies.

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Mesh:

Year:  2008        PMID: 18509743      PMCID: PMC2682766          DOI: 10.1007/s10545-008-0821-1

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN: 0141-8955            Impact factor:   4.982


  14 in total

1.  Alpha-L-iduronidase deficiency in a cat: a model of mucopolysaccharidosis I.

Authors:  M E Haskins; P F Jezyk; R J Desnick; S K McDonough; D F Patterson
Journal:  Pediatr Res       Date:  1979-11       Impact factor: 3.756

2.  Enzyme replacement therapy in feline mucopolysaccharidosis I.

Authors:  E D Kakkis; E Schuchman; X He; Q Wan; S Kania; S Wiemelt; C W Hasson; T O'Malley; M A Weil; G A Aguirre; D E Brown; M E Haskins
Journal:  Mol Genet Metab       Date:  2001-03       Impact factor: 4.797

3.  Effects of bone marrow transplantation on the cardiovascular abnormalities in canine mucopolysaccharidosis VII.

Authors:  C Sammarco; M Weil; C Just; S Weimelt; C Hasson; T O'Malley; S M Evans; P Wang; M L Casal; J Wolfe; M Haskins
Journal:  Bone Marrow Transplant       Date:  2000-06       Impact factor: 5.483

4.  Animal models of mucopolysaccharidosis.

Authors:  M E Haskins; P F Jezyk; R J Desnick; M M McGovern; D T Vine; D F Patterson
Journal:  Prog Clin Biol Res       Date:  1982

5.  Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig.

Authors:  Katherine P Ponder; Baomei Wang; Ping Wang; Xiucui Ma; Ramin Herati; Bin Wang; Karyn Cullen; Patty O'Donnell; N Matthew Ellinwood; Anne Traas; Tina M Primeau; Mark E Haskins
Journal:  Mol Ther       Date:  2006-05-12       Impact factor: 11.454

6.  Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs.

Authors:  Katherine Parker Ponder; John R Melniczek; Lingfei Xu; Margaret A Weil; Thomas M O'Malley; Patricia A O'Donnell; Van W Knox; Gustavo D Aguirre; Hamutal Mazrier; N Matthew Ellinwood; Meg Sleeper; Albert M Maguire; Susan W Volk; Robert L Mango; Jean Zweigle; John H Wolfe; Mark E Haskins
Journal:  Proc Natl Acad Sci U S A       Date:  2002-09-13       Impact factor: 11.205

7.  Clinical and biochemical study of 28 patients with mucopolysaccharidosis type VI.

Authors:  A C M M Azevedo; I V Schwartz; L Kalakun; S Brustolin; M G Burin; A P C Beheregaray; S Leistner; C Giugliani; M Rosa; P Barrios; D Marinho; P Esteves; E Valadares; R Boy; D Horovitz; P Mabe; L C S da Silva; I C N de Souza; M Ribeiro; A M Martins; D Palhares; C A Kim; R Giugliani
Journal:  Clin Genet       Date:  2004-09       Impact factor: 4.438

Review 8.  Gene therapy for lysosomal storage diseases: the lessons and promise of animal models.

Authors:  N Matthew Ellinwood; Charles H Vite; Mark E Haskins
Journal:  J Gene Med       Date:  2004-05       Impact factor: 4.565

9.  Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII.

Authors:  M M Sleeper; B Fornasari; N M Ellinwood; M A Weil; J Melniczek; T M O'Malley; C D Sammarco; L Xu; K P Ponder; M E Haskins
Journal:  Circulation       Date:  2004-08-02       Impact factor: 29.690

10.  The pathology of the feline model of mucopolysaccharidosis I.

Authors:  M E Haskins; G D Aguirre; P F Jezyk; R J Desnick; D F Patterson
Journal:  Am J Pathol       Date:  1983-07       Impact factor: 4.307

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  13 in total

1.  Pathogenesis of aortic dilatation in mucopolysaccharidosis VII mice may involve complement activation.

Authors:  Guilherme Baldo; Susan Wu; Ruth A Howe; Meera Ramamoothy; Russell H Knutsen; Jiali Fang; Robert P Mecham; Yuli Liu; Xiaobo Wu; John P Atkinson; Katherine P Ponder
Journal:  Mol Genet Metab       Date:  2011-08-24       Impact factor: 4.797

2.  Cardiac disease in mucopolysaccharidosis type I attributed to catecholaminergic and hemodynamic deficiencies.

Authors:  Nathan J Palpant; Fikru B Bedada; Brandon Peacock; Bruce R Blazar; Joseph M Metzger; Jakub Tolar
Journal:  Am J Physiol Heart Circ Physiol       Date:  2010-11-12       Impact factor: 4.733

3.  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

4.  Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.

Authors:  Katherine P Ponder; Thomas M O'Malley; Ping Wang; Patricia A O'Donnell; Anne M Traas; Van W Knox; Gustavo A Aguirre; N Matthew Ellinwood; Jason A Metcalf; Bin Wang; Emma J Parkinson-Lawrence; Meg M Sleeper; Doug A Brooks; John J Hopwood; Mark E Haskins
Journal:  Mol Ther       Date:  2012-03-06       Impact factor: 11.454

5.  Carotid intima-media thickness is increased in patients with treated mucopolysaccharidosis types I and II, and correlates with arterial stiffness.

Authors:  Raymond Y Wang; Elizabeth A Braunlin; Kyle D Rudser; Donald R Dengel; Andrea M Metzig; Kelly K Covault; Lynda E Polgreen; Elsa Shapiro; Julia Steinberger; Aaron S Kelly
Journal:  Mol Genet Metab       Date:  2013-11-12       Impact factor: 4.797

6.  Upregulation of elastase activity in aorta in mucopolysaccharidosis I and VII dogs may be due to increased cytokine expression.

Authors:  Jason A Metcalf; Bruce Linders; Susan Wu; Paul Bigg; Patricia O'Donnell; Meg M Sleeper; Michael P Whyte; Mark Haskins; Katherine P Ponder
Journal:  Mol Genet Metab       Date:  2009-12-11       Impact factor: 4.797

7.  Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.

Authors:  Gabriella Cotugno; Patrizia Annunziata; Alessandra Tessitore; Thomas O'Malley; Anita Capalbo; Armida Faella; Rosa Bartolomeo; Patricia O'Donnell; Ping Wang; Fabio Russo; Meg M Sleeper; Van W Knox; Steven Fernandez; Leah Levanduski; John Hopwood; Elvira De Leonibus; Mark Haskins; Alberto Auricchio
Journal:  Mol Ther       Date:  2010-11-30       Impact factor: 11.454

8.  Aortic Root Dilatation in Mucopolysaccharidosis I-VII.

Authors:  Meena Bolourchi; Pierangelo Renella; Raymond Y Wang
Journal:  Int J Mol Sci       Date:  2016-11-29       Impact factor: 5.923

9.  Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI).

Authors:  Paola Saccone; Gabriella Cotugno; Fabio Russo; Rosa Mastrogiacomo; Alessandra Tessitore; Alberto Auricchio; Elvira De Leonibus
Journal:  Sci Rep       Date:  2014-01-10       Impact factor: 4.379

10.  Mucopolysaccharidosis VI in cats - clarification regarding genetic testing.

Authors:  Leslie A Lyons; Robert A Grahn; Francesca Genova; Michela Beccaglia; John J Hopwood; Maria Longeri
Journal:  BMC Vet Res       Date:  2016-07-02       Impact factor: 2.741

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