High frequency of W1327X mutation in glycogen storage disease type III patients from central Tunisia

被引:5
作者
Cherif, Wafa [1 ,2 ]
Ben Rhouma, Faten [1 ]
Messai, Habib [1 ]
Mili, Amira [3 ]
Gribaa, Moez [4 ]
Kefi, Rym [1 ]
Ayadi, Abdelkarim [5 ]
Boughamoura, Lamia [6 ]
Chemli, Jelel [7 ]
Saad, Ali [4 ]
Kaabachi, Naziha [8 ]
Sfar, Mohamed Tahar [5 ]
Ben Dridi, Marie-Francoise [2 ]
Tebib, Neji [2 ]
Abdelhak, Sonia [1 ]
机构
[1] Pasteur Inst Tunis, Lab Biomed Genom & Oncogenet LR11IPT05, Tunis 1002, Tunisia
[2] La Rabta Hosp, Dept Pediat, Inherited Metab Dis Unit, Tunis, Tunisia
[3] Med Univ, Biochem Lab, Sousse, Tunisia
[4] Farhat Hached Hosp, Cytogenet Lab, Sousse, Tunisia
[5] Mahdia Hosp, Dept Pediat, Mahdia 5100, Tunisia
[6] Farhat Hached Hosp, Dept Pediat, Sousse 4031, Tunisia
[7] Sahloul Hosp, Dept Pediat, Sousse, Tunisia
[8] La Rabta Hosp, Biochem Lab, Tunis 1007, Tunisia
关键词
glycogen storage disease type III; molecular diagnosis; Tunisia; founder effect; W1327X mutation; DEBRANCHING ENZYME; DEFICIENCY; FEATURES; TURKEY;
D O I
10.1684/abc.2012.0766
中图分类号
R446 [实验室诊断]; R-33 [实验医学、医学实验];
学科分类号
1001 ;
摘要
Glycogen storage disease type III (GSD III) is an autosomal recessive disorder caused by the deficiency of glycogen debranching enzyme (AGL). It is characterized by hepatomegaly, progressive myopathy, cardiomyopathy and fasting hypoglycemia. Several mutations in AGL gene have been described in different populations. The W1327X mutation was reported in one Tunisian patient resident in Italy. We looked in this report to determine the frequency of W1327X mutation among Tunisian patients. The W1327X mutation was screening in 26 GSD III patients originated from various geographic locations in Tunisia. The sequence analysis revealed that among nine patients carried the W1327X mutation. Eight of them were from six unrelated families and they were originated from Mahdia (centre of Tunisia) suggesting the existence of a founder effect in this region. Taking into account historical migratory waves, screening for this mutation should be performed in priority for molecular diagnosis confirmation of GSD III in North African populations.
引用
收藏
页码:648 / 650
页数:3
相关论文
共 11 条
[1]   Molecular features of 23 patients with glycogen storage disease type III in Turkey: a novel mutation p.R1147G associated with isolated glucosidase deficiency, along with 9 AGL mutations [J].
Aoyama, Yoshiko ;
Ozer, Isil ;
Demirkol, Mubeccel ;
Ebara, Tetsu ;
Murase, Toshio ;
Podskarbi, Teodor ;
Shin, Yoon S. ;
Gokcay, Gulden ;
Okubo, Minoru .
JOURNAL OF HUMAN GENETICS, 2009, 54 (11) :681-686
[2]   Mutation spectrum of glycogen storage disease type Ia in Tunisia: Implication for molecular diagnosis [J].
Barkaoui, E. ;
Cherif, W. ;
Tebib, N. ;
Charfeddine, C. ;
Ben Rhouma, F. ;
Azzouz, H. ;
Ben Chehida, A. ;
Monastiri, K. ;
Chemli, J. ;
Amri, F. ;
Ben Turkia, H. ;
Abdelmoula, M. S. ;
Kaabachi, N. ;
Abdelhak, S. ;
Ben Dridi, M. F. .
JOURNAL OF INHERITED METABOLIC DISEASE, 2007, 30 (06) :989-989
[3]   GLYCOGEN DEBRANCHING ENZYME DEFICIENCY - LONG-TERM STUDY OF SERUM ENZYME-ACTIVITIES AND CLINICAL-FEATURES [J].
COLEMAN, RA ;
WINTER, HS ;
WOLF, B ;
CHEN, YT .
JOURNAL OF INHERITED METABOLIC DISEASE, 1992, 15 (06) :869-881
[4]   Molecular characterization of Egyptian patients with glycogen storage disease type IIIa [J].
Endo, Y ;
Fateen, E ;
Aoyama, Y ;
Horinishi, A ;
Ebara, T ;
Murase, T ;
Shin, YS ;
Okubo, M .
JOURNAL OF HUMAN GENETICS, 2005, 50 (10) :538-542
[5]   Molecular analysis of the AGL gene: heterogeneity of mutations in patients with glycogen storage disease type III from Germany, Canada, Afghanistan, Iran, and Turkey [J].
Endo, Yoriko ;
Horinishi, Asako ;
Vorgerd, Matthias ;
Aoyama, Yoshiko ;
Ebara, Tetsu ;
Murase, Toshio ;
Odawara, Masato ;
Podskarbi, Teodor ;
Shin, Yoon S. ;
Okubo, Minoru .
JOURNAL OF HUMAN GENETICS, 2006, 51 (11) :958-963
[6]   Molecular Characterisation of GSD III Subjects and Identification of Six Novel Mutations in AGL [J].
Lucchiari, S. ;
Donati, M. A. ;
Parini, R. ;
Melis, D. ;
Gatti, R. ;
Bresolin, N. ;
Scarlato, G. ;
Comi, G. P. .
HUMAN MUTATION, 2002, 20 (06) :480
[7]   Identification of the catalytic residues of bifunctional glycogen debranching enzyme [J].
Nakayama, A ;
Yamamoto, K ;
Tabata, S .
JOURNAL OF BIOLOGICAL CHEMISTRY, 2001, 276 (31) :28824-28828
[8]   Clinicopathological Analysis of the Homozygous p.W1327X AGL Mutation in Glycogen Storage Disease Type 3 [J].
Schoser, Benedikt ;
Glaeser, Dieter ;
Mueller-Hoecker, Josef .
AMERICAN JOURNAL OF MEDICAL GENETICS PART A, 2008, 146A (22) :2911-2915
[9]  
Shen J.-J., 2002, Current Molecular Medicine (Hilversum), V2, P167, DOI 10.2174/1566524024605752
[10]   DELETION OF ARGININE (608) IN ACID SPHINGOMYELINASE IS THE PREVALENT MUTATION AMONG NIEMANN-PICK DISEASE TYPE-B PATIENTS FROM NORTHERN AFRICA [J].
VANIER, MT ;
FERLINZ, K ;
ROUSSON, R ;
DUTHEL, S ;
LOUISOT, P ;
SANDHOFF, K ;
SUZUKI, K .
HUMAN GENETICS, 1993, 92 (04) :325-330