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Brandl, Caroline ; Schulz, Heidi ; Charbel Issa, Peter ; Birtel, Johannes ; Bergholz, Richard ; Lange, Clemens ; Dahlke, Claudia ; Zobor, Ditta ; Weber, Bernhard ; Stöhr, Heidi

Mutations in the Genes for Interphotoreceptor Matrix Proteoglycans, IMPG1 and IMPG2, in Patients with Vitelliform Macular Lesions

Brandl, Caroline , Schulz, Heidi, Charbel Issa, Peter, Birtel, Johannes, Bergholz, Richard, Lange, Clemens, Dahlke, Claudia, Zobor, Ditta, Weber, Bernhard und Stöhr, Heidi (2017) Mutations in the Genes for Interphotoreceptor Matrix Proteoglycans, IMPG1 and IMPG2, in Patients with Vitelliform Macular Lesions. Genes 8 (170), S. 1-14.

Veröffentlichungsdatum dieses Volltextes: 05 Dez 2017 14:42
Artikel
DOI zum Zitieren dieses Dokuments: 10.5283/epub.36413


Zusammenfassung

A significant portion of patients diagnosed with vitelliform macular dystrophy (VMD) do not carry causative mutations in the classic VMD genes BEST1 or PRPH2. We therefore performed a mutational screen in a cohort of 106 BEST1/PRPH2-negative VMD patients in two genes encoding secreted interphotoreceptor matrix proteoglycans-1 and -2 (IMPG1 and IMPG2). We identified two novel mutations in IMPG1 in ...

A significant portion of patients diagnosed with vitelliform macular dystrophy (VMD) do not carry causative mutations in the classic VMD genes BEST1 or PRPH2. We therefore performed a mutational screen in a cohort of 106 BEST1/PRPH2-negative VMD patients in two genes encoding secreted interphotoreceptor matrix proteoglycans-1 and -2 (IMPG1 and IMPG2). We identified two novel mutations in IMPG1 in two simplex VMD cases with disease onset in their early childhood, a heterozygous p.(Leu238Pro) missense mutation and a homozygous c.807 + 5G > A splice site mutation. The latter induced partial skipping of exon 7 of IMPG1 in an in vitro splicing assay. Furthermore, we found heterozygous mutations including three stop [p.(Glu226*), p.(Ser522*), p.(Gln856*)] and five missense mutations [p.(Ala243Pro), p.(Gly1008Asp), p.(Phe1016Ser), p.(Tyr1042Cys), p.(Cys1077Phe)] in the IMPG2 gene, one of them, p.(Cys1077Phe), previously associated with VMD. Asymptomatic carriers of the p.(Ala243Pro) and p.(Cys1077Phe) mutations show subtle foveal irregularities that could characterize a subclinical stage of disease. Taken together, our results provide further evidence for an involvement of dominant and recessive mutations in IMPG1 and IMPG2 in VMD pathology. There is a remarkable similarity in the clinical appearance of mutation carriers, presenting with bilateral, central, dome-shaped foveal accumulation of yellowish material with preserved integrity of the retinal pigment epithelium (RPE). Clinical symptoms tend to be more severe for IMPG1 mutations.



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Details

DokumentenartArtikel
Titel eines Journals oder einer ZeitschriftGenes
Verlag:MDPI
Ort der Veröffentlichung:BASEL
Band:8
Nummer des Zeitschriftenheftes oder des Kapitels:170
Seitenbereich:S. 1-14
Datum23 Juni 2017
InstitutionenMedizin > Lehrstuhl für Augenheilkunde
Medizin > Lehrstuhl für Humangenetik
Medizin > Institut für Epidemiologie und Präventivmedizin
Identifikationsnummer
WertTyp
10.3390/genes8070170DOI
Stichwörter / KeywordsRETINITIS-PIGMENTOSA; DYSTROPHY; PROTEIN; vitelliform macular dystrophy; IMPG1; IMPG2; interphotoreceptor matrix; optical coherence tomography
Dewey-Dezimal-Klassifikation600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin
StatusVeröffentlicht
BegutachtetJa, diese Version wurde begutachtet
An der Universität Regensburg entstandenJa
URN der UB Regensburgurn:nbn:de:bvb:355-epub-364134
Dokumenten-ID36413

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