| Literature DB >> 18509892 |
Uluç Yiş1, Stefano Pepe, Semra Hiz Kurul, Andrea Ballabio, Maria Pia Cosma, Eray Dirik.
Abstract
Multiple sulfatase deficiency (MSD) is an inherited lysosomal storage disease that affects post-translational activation of all of the sulfatases. Since biochemical and clinical findings are variable, the diagnosis is difficult in most of the cases. Missense, nonsense, microdeletion and splicing mutations in SUMF1 gene were found in all of the MSD patients analyzed. Here, we present clinical findings of two consanguineous patients with multiple sulfatase deficiency. They were found to be homozygous for a novel missense mutation c.739G > C causing a p.G247R amino acid substitution in the SUMF1 protein.Entities:
Mesh:
Substances:
Year: 2008 PMID: 18509892 DOI: 10.1016/j.braindev.2007.10.007
Source DB: PubMed Journal: Brain Dev ISSN: 0387-7604 Impact factor: 1.961