A new congenital disorder of glycosylation caused by a mutation in SSR4, the signal sequence receptor 4 protein of the TRAP complex.

TitleA new congenital disorder of glycosylation caused by a mutation in SSR4, the signal sequence receptor 4 protein of the TRAP complex.
Publication TypeJournal Article
Year of Publication2014
AuthorsLosfeld, MEstelle, Ng, BG, Kircher, M, Buckingham, KJ, Turner, EH, Eroshkin, A, Smith, JD, Shendure, J, Nickerson, DA, Bamshad, MJ, Freeze, HH
Corporate AuthorsUniversity of Washington Center for Mendelian Genomics
JournalHum Mol Genet
Volume23
Issue6
Pagination1602-5
Date Published2014 Mar 15
ISSN1460-2083
KeywordsAdolescent, Calcium-Binding Proteins, Cells, Cultured, Chromosomes, Human, X, Congenital Disorders of Glycosylation, Glycosylation, Humans, Male, Membrane Glycoproteins, Point Mutation, Receptors, Cytoplasmic and Nuclear, Receptors, Peptide
Abstract

Nearly 50 congenital disorders of glycosylation (CDG) are known, but many patients biochemically diagnosed with CDG do not have mutations in known genes. Here, we describe a 16-year-old male who was born with microcephaly, developed intellectual disability, gastroesophageal reflux and a seizure disorder. We identified a de novo variant in the X-linked SSR4 gene which encodes a protein of the heterotetrameric translocon-associated protein (TRAP) complex. The c.316delT causes a p.F106Sfs*53 in SSR4 and also reduces expression of other TRAP complex proteins. The glycosylation marker Glyc-ER-GFP was used to confirm the underglycosylation in fibroblasts from the patient. Overexpression of the wild-type SSR4 allele partially restores glycosylation of the marker and of the other members of the TRAP complex. This is the first evidence that the TRAP complex, which binds to the oligosaccharyltransferase complex, is directly involved in N-glycosylation.

DOI10.1093/hmg/ddt550
Alternate JournalHum. Mol. Genet.
PubMed ID24218363
PubMed Central IDPMC3929095
Grant ListU54 HG006493 / HG / NHGRI NIH HHS / United States
UM1 HG006493 / HG / NHGRI NIH HHS / United States
R01DK55615 / DK / NIDDK NIH HHS / United States