Regulation of N-Acetylgalactosamine 4-Sulfatase Expression in Retrovirus-Transduced Feline Mucopolysaccharidosis Type VI Muscle Cells

Yogalingam, Gouri; Muller, Vivienne; Hopwood, John J.; Anson, Donald S.
March 1999
DNA & Cell Biology;Mar1999, Vol. 18 Issue 3, p187
Academic Journal
As a preliminary step toward muscle-mediated gene therapy in the mucopolysaccharidosis (MPS) type VI cat, we have analyzed the transcriptional regulation of feline N-acetylgalactosamine 4-sulfatase (f4S) gene expression from various retroviral constructs in primary cultures of muscle cells. Two retroviral constructs were made containing the f4S cDNA under the transcriptional control of the human polypeptide chain-elongation factor 1 alpha (EF1 alpha) gene promoter or the cytomegalovirus (CMV) immediate-early promoter. Two further retroviral constructs were made with the murine muscle creatine kinase (mck) enhancer sequence upstream of the internal promoter. Virus made from each construct was used to transduce feline MPS VI myoblasts. The mck enhancer significantly upregulated f4S gene expression from both the EF1 alpha promoter and the CMV promoter in transduced myoblasts and in differentiated myofibers. The highest level of 4S activity was observed in myoblasts and myofibers transduced with the retroviral construct L mckcmv4S, in which the f4S gene is under the transcriptional regulation of the mck enhancer and CMV immediate-early promoter. L mckcmv4S-transduced myofibers demonstrated correction of glycosaminoglycan storage and contained a 58-fold elevated level of 4S activity compared with normal myofibers. Recombinant f4S secreted from L mckcmv4S-transduced myofibers was endocytosed by feline MPS VI myofibers, leading to correction of the biochemical storage phenotype.


Related Articles

  • A model for the mechanical events during the contraction of an isolated heart muscle cell. Muntingh, L.; Hanekom, J.J. // South African Journal of Science;Jun97, Vol. 93 Issue 6, p292 

    Introduces a model used to describe the dynamics of specific anatomical structures within a myocyte. Combination of anatomical structures with the same physiology; Exhibition of same rate of contraction, maximum amplitude of contraction and step response characteristics observed in...

  • PHASE 3 TRIAL/STUDY OF ALDURAZYME FOR MPS I PROMISING.  // Worldwide Biotech;Aug2002, Vol. 14 Issue 8, p8 

    Reports on the results of the clinical trial of Aldurazyme, an investigational enzyme replacement therapy for patients with mucopolysaccharidosis I.

  • The mild form of mucopolysaccharidosis type I (Scheie syndrome) is associated with increased ascending aortic stiffness. Nemes, Attila; Timmermans, Remco G. M.; WIlson, J. H. Paul; Soliman, Osama I. I.; Krenning, Boudewjim J.; ten Cate, Folkert J.; Geleijnse, Marcel L. // Heart & Vessels;Jul2008, Vol. 23 Issue 4, p293 

    A correction to the article "The mild form of mucopolysaccharidosis type I (Scheie syndrome) is associated with increased ascending aortic stiffness" that was published in the previous issue is presented .

  • glucuronidase deficiency disease.  // Taber's Cyclopedic Medical Dictionary (2009);2009, Issue 21, p967 

    An encyclopedia entry for "glucoronidase deficiency disease," which refers to mucopolysaccharidosis, is presented.

  • lipochondrodystrophy.  // Taber's Cyclopedic Medical Dictionary (2009);2009, Issue 21, p1348 

    A definition of the term "lipochondrodystrophy," which means mucopolysaccharidosis, is presented.

  • Maroteaux-Lamy syndrome.  // Taber's Cyclopedic Medical Dictionary (2009);2009, Issue 21, p1405 

    A definition of the term "Maroteaux-Lamy syndrome," which refers to Mucopolysaccharidosis VI, is presented.

  • MPS.  // Taber's Cyclopedic Medical Dictionary (2009);2009, Issue 21, p1500 

    An encyclopedia entry for the medical acronym "MPS," which refers to mucopolysaccharidosis and mononuclear phagocytic system, is presented.

  • Scheie's syndrome.  // Taber's Cyclopedic Medical Dictionary (2009);2009, Issue 21, p2078 

    A definition of the term "Scheie's syndrome," which refers to mucopolysaccharidosis, is presented.

  • Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome) in the pre-Columbian culture of Colombia. Pachajoa, Harry; Armando Rodriguez, Carlos // Colombia Medica;Apr-Jun2014, Vol. 45 Issue 2, p85 

    Mucopolysaccharidosis type VI or Maroteaux Lamy syndrome is an autosomal recessive lysosomal storage disorder resulting from a deficiency of arylsulfatase B, the clinical features include short stature, hepatosplenomegaly, dysostosis multiplex, stiff joints, corneal clouding, cardiac...


Read the Article


Sorry, but this item is not currently available from your library.

Try another library?
Sign out of this library

Other Topics