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  • WJPPS JANUARY ISSUE PUBLISHED
  • JANUARY 2019 Issue has been successfully launched on 1 January 2019

  • WJPPS Impact Factor
  • Its our Pleasure to Inform you that WJPPS Impact Factor has been increased from 6.647 to 7.421 due to high quality Publication at International Level

  • ICV
  • WJPPS Rank with Index Copernicus Value 84.65 due to high reputation at International Level

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    • WJPPS  Invited to submit your valuable manuscripts for Coming Issue.
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  • WJPPS introducing updated version of OSTS (online submission and tracking system), which have dedicated control panel for both author and reviewer. Using this control panel author can submit manuscript
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Abstract

A REVIEW ON LYSOSOMAL STORAGE DISORDERS

Dr. K. N. S. V. Lakshmi*, A. Ankarao and A. Seetha Devi

ABSTRACT

Lysosomal storage diseases are a group of rare, inborn, metabolic errors characterized by deficiencies in normal lysosomal function and by intralysosomal accumulation of undegraded substrates. Classically, lysosomal storage diseases encompassed only enzyme deficiencies of the lysosomal hydrolases. More recently, the concept of lysosomal storage disease has been expanded to include deficiencies or defects in proteins necessary for the normal post-translational modification of lysosomal enzymes (which themselves are often glycoproteins), activator proteins, or proteins important for proper intracellular trafficking between the lysosome and other intracellular compartments. The past 25 years have been characterized by remarkable progress in the treatment of these diseases and by the development of multiple therapeutic approaches. These approaches include strategies aimed at increasing the residual activity of a missing enzyme (enzyme replacement therapy, hematopoietic stem cell transplantation, pharmacological chaperone therapy and gene therapy) and approaches based on reducing the flux of substrates to lysosomes.

Keywords: Lysosomal, intralysosomal.


[Full Text Article]

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