Abetalipoproteinemia (ABL; OMIM 200100) is a rare autosomal recessive disease that affects the absorption of dietary fats and fat soluble vitamins. Here, we describe the clinical and genetic characteristics of three patients with ABL. Two patients (patients 1 and 2) who were carriers of the c.398-399delAA mutation (previously known mutation) had developmental delay and hepatic steatosis developed at the age of five in patient 1. Patient 3 was the carrier of a novel mutation (g.10886-10902delAAGgtaagtttgtgttg in intron 3 and c.506A>T exon 5) in microsomal triglyceride transfer protein (MTP) gene and had hepatic steatosis.

Gündüz, M., Özaydın, E., Atar, M., Koç, N., Kırsaçlıoğlu, C., Köse, G., et al. (2016). Microsomal triglyceride transfer protein gene mutations in Turkish children: A novel mutation and clinical follow up. INDIAN JOURNAL OF GASTROENTEROLOGY, 35(3), 236-241 [10.1007/s12664-016-0654-z].

Microsomal triglyceride transfer protein gene mutations in Turkish children: A novel mutation and clinical follow up

CEFALU', Angelo Baldassare;AVERNA, Maurizio;
2016-01-01

Abstract

Abetalipoproteinemia (ABL; OMIM 200100) is a rare autosomal recessive disease that affects the absorption of dietary fats and fat soluble vitamins. Here, we describe the clinical and genetic characteristics of three patients with ABL. Two patients (patients 1 and 2) who were carriers of the c.398-399delAA mutation (previously known mutation) had developmental delay and hepatic steatosis developed at the age of five in patient 1. Patient 3 was the carrier of a novel mutation (g.10886-10902delAAGgtaagtttgtgttg in intron 3 and c.506A>T exon 5) in microsomal triglyceride transfer protein (MTP) gene and had hepatic steatosis.
2016
Settore MED/09 - Medicina Interna
Gündüz, M., Özaydın, E., Atar, M., Koç, N., Kırsaçlıoğlu, C., Köse, G., et al. (2016). Microsomal triglyceride transfer protein gene mutations in Turkish children: A novel mutation and clinical follow up. INDIAN JOURNAL OF GASTROENTEROLOGY, 35(3), 236-241 [10.1007/s12664-016-0654-z].
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10447/183596
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