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-acetyl-galactosamine-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.
journal_name
Gene Therjournal_title
Gene therapyauthors
Ohashi T,Matalon R,Barranger JA,Eto Ysubject
Has Abstractpub_date
1995-08-01 00:00:00pages
363-8issue
6eissn
0969-7128issn
1476-5462journal_volume
2pub_type
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