TY - JOUR
T1 - Key features and clinical variability of COG6-CDG
AU - Rymen, Daisy
AU - Winter, Julia
AU - Van Hasselt, Peter M
AU - Jaeken, Jaak
AU - Kasapkara, Cigdem
AU - Gokçay, Gulden
AU - Haijes, Hanneke
AU - Goyens, Philippe
AU - Tokatli, Aysegul
AU - Thiel, Christian
AU - Bartsch, Oliver
AU - Hecht, Jochen
AU - Krawitz, Peter
AU - Prinsen, Hubertus C M T
AU - Mildenberger, Eva
AU - Matthijs, Gert
AU - Kornak, Uwe
N1 - Copyright © 2015. Published by Elsevier Inc.
PY - 2015/11
Y1 - 2015/11
N2 - The conserved oligomeric Golgi (COG) complex consists of eight subunits and plays a crucial role in Golgi trafficking and positioning of glycosylation enzymes. Mutations in all COG subunits, except subunit 3, have been detected in patients with congenital disorders of glycosylation (CDG) of variable severity. So far, 3 families with a total of 10 individuals with biallelic COG6 mutations have been described, showing a broad clinical spectrum. Here we present 7 additional patients with 4 novel COG6 mutations. In spite of clinical variability, we delineate the core features of COG6-CDG i.e. liver involvement (9/10), microcephaly (8/10), developmental disability (8/10), recurrent infections (7/10), early lethality (6/10), and hypohidrosis predisposing for hyperthermia (6/10) and hyperkeratosis (4/10) as ectodermal signs. Regarding all COG6-related disorders a genotype-phenotype correlation can be discerned ranging from deep intronic mutations found in Shaheen syndrome as the mildest form to loss-of-function mutations leading to early lethal CDG phenotypes. A comparison with other COG deficiencies suggests ectodermal changes to be a hallmark of COG6-related disorders. Our findings aid clinical differentiation of this complex group of disorders and imply subtle functional differences between the COG complex subunits.
AB - The conserved oligomeric Golgi (COG) complex consists of eight subunits and plays a crucial role in Golgi trafficking and positioning of glycosylation enzymes. Mutations in all COG subunits, except subunit 3, have been detected in patients with congenital disorders of glycosylation (CDG) of variable severity. So far, 3 families with a total of 10 individuals with biallelic COG6 mutations have been described, showing a broad clinical spectrum. Here we present 7 additional patients with 4 novel COG6 mutations. In spite of clinical variability, we delineate the core features of COG6-CDG i.e. liver involvement (9/10), microcephaly (8/10), developmental disability (8/10), recurrent infections (7/10), early lethality (6/10), and hypohidrosis predisposing for hyperthermia (6/10) and hyperkeratosis (4/10) as ectodermal signs. Regarding all COG6-related disorders a genotype-phenotype correlation can be discerned ranging from deep intronic mutations found in Shaheen syndrome as the mildest form to loss-of-function mutations leading to early lethal CDG phenotypes. A comparison with other COG deficiencies suggests ectodermal changes to be a hallmark of COG6-related disorders. Our findings aid clinical differentiation of this complex group of disorders and imply subtle functional differences between the COG complex subunits.
KW - COG6
KW - CDG
KW - Conserved oligomeric Golgi complex
KW - Congenital disorder of glycosylation
UR - https://www.scopus.com/pages/publications/84948716414
U2 - 10.1016/j.ymgme.2015.07.003
DO - 10.1016/j.ymgme.2015.07.003
M3 - Article
C2 - 26260076
SN - 1096-7192
VL - 116
SP - 163
EP - 170
JO - Molecular Genetics and Metabolism
JF - Molecular Genetics and Metabolism
IS - 3
ER -