Hyaluronidase increases the biodistribution of acid α-1,4 glucosidase in the muscle of Pompe disease mice

An approach to enhance the efficacy of enzyme replacement therapy

Reuben Matalon, Sankar Surendran, Gerald Campbell, Kimberlee Michals-Matalon, Stephen K. Tyring, James Grady, Seng Cheng, Edward Kaye

Research output: Contribution to journalArticle

6 Citations (Scopus)

Abstract

Pompe disease (glycogen storage disease type II) is a glycogen storage disease caused by a deficiency of the lysosomal enzyme, acid maltase/acid α-1,4 glucosidase (GAA). Deficiency of the enzyme leads primarily to intra-lysosomal glycogen accumulation, primarily in cardiac and skeletal muscles, due to the inability of converting glycogen into glucose. Enzyme replacement therapy (ERT) has been applied to replace the deficient enzyme and to restore the lost function. However, enhancing the enzyme activity to the muscle following ERT is relatively insufficient. In order to enhance GAA activity into the muscle in Pompe disease, efficacy of hyaluronidase (hyase) was examined in the heart, quadriceps, diaphragm, kidney, and brain of mouse model of Pompe disease. Administration of hyase 3000 U/mouse (intravenous) i.v. or i.p. (intraperitoneal) and 10 min later recombinant human GAA (rhGAA) 20 mg/kg i.v. showed more GAA activity in hyase i.p. injected mice compared to those mice injected with hyase via i.v. Injection of low dose of hyase (3000 U/mouse) or high dose of hyase (10,000 U/mouse) i.p. and 20 min or 60 min later 20 mg/kg rhGAA i.v. increased GAA activity into the heart, diaphragm, kidney, and quadriceps compared to hyase untreated mice. These studies suggest that hyase enhances penetration of enzyme into the tissues including muscle during ERT and therefore hyase pretreatment may be important in treating Pompe disease.

Original languageEnglish (US)
Pages (from-to)783-787
Number of pages5
JournalBiochemical and Biophysical Research Communications
Volume350
Issue number3
DOIs
StatePublished - Nov 24 2006

Fingerprint

Glycogen Storage Disease Type II
Glucosidases
Enzyme Replacement Therapy
Hyaluronoglucosaminidase
Muscle
Muscles
Acids
Enzymes
Glycogen
Diaphragms
Diaphragm
Glycogen Storage Disease
Kidney
alpha-Glucosidases
Enzyme activity
Brain
Myocardium
Skeletal Muscle
Tissue
Glucose

Keywords

  • Acid α-1,4 glucosidase
  • Acid maltase deficiency
  • Enzyme replacement therapy
  • GAA
  • Pompe disease
  • Skeletal muscle

ASJC Scopus subject areas

  • Biochemistry
  • Biophysics
  • Molecular Biology

Cite this

Hyaluronidase increases the biodistribution of acid α-1,4 glucosidase in the muscle of Pompe disease mice : An approach to enhance the efficacy of enzyme replacement therapy. / Matalon, Reuben; Surendran, Sankar; Campbell, Gerald; Michals-Matalon, Kimberlee; Tyring, Stephen K.; Grady, James; Cheng, Seng; Kaye, Edward.

In: Biochemical and Biophysical Research Communications, Vol. 350, No. 3, 24.11.2006, p. 783-787.

Research output: Contribution to journalArticle

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