Overexpression of arylsulfatase A gene in fibroblasts from metachromatic leukodystrophy patients does not induce a new phenotype

T. Ohashi, R. Matalon, J. A. Barranger, Y. Eto

Research output: Contribution to journalArticle

8 Scopus citations

Abstract

We tested the influence of overexpression of arylsulfatase A (ASA) on the activity of other sulfatases in fibroblasts from patients with metachromatic leukodystrophy (MLD). We demonstrated that the overexpression of ASA reduces the activity of various sulfatases by a small amount but does not induce an accumulation of glycosaminoglycan. Our results indicate that influence of ASA overexpression on other sulfatases is different from that of N-acetylgalactosamine-4-sulfatase overexpression reported by Anson et al. We conclude that gene therapy for MLD based on the transfer of a normal ASA gene to mutant cells will be feasible because the overexpression of ASA peptides in cells does not lead to profound deficiency of other sulfatases or result in a new phenotype.

Original languageEnglish (US)
Pages (from-to)363-368
Number of pages6
JournalGene Therapy
Volume2
Issue number6
StatePublished - Jan 1 1995
Externally publishedYes

Keywords

  • arylsulfatase A
  • gene therapy
  • glycosaminoglycan
  • metachromatic leukodystrophy
  • multiple sulfatase deficiency
  • sulfatase

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

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