Literature DB >> 7945303

Deletion of exon 18 is a frequent mutation in glycogen storage disease type II.

M Van der Kraan1, M A Kroos, M Joosse, A G Bijvoet, M P Verbeet, W J Kleijer, A J Reuser.   

Abstract

An abnormal 2.3 kb SacI fragment of the human lysosomal alpha-glucosidase gene (GAA) was identified in patients with glycogen storage disease type II. The fragment results from deletion of exon 18 and adjacent parts of intron 17 and 18. The borders of the deletion are marked by the occurrence of an eight nucleotide long tandem repeat (AGGGGCCG) which is apparently instrumental in the mutation event. The exon 18 deletion was demonstrated in 10 out of 39 patients from Europe (all hetero-allelic) and is so far the most common mutation in this disease (allele frequency among patients is 0.13).

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Year:  1994        PMID: 7945303     DOI: 10.1006/bbrc.1994.2360

Source DB:  PubMed          Journal:  Biochem Biophys Res Commun        ISSN: 0006-291X            Impact factor:   3.575


  14 in total

1.  Molecular diagnosis of German patients with late-onset glycogen storage disease type II.

Authors:  P R Joshi; D Gläser; S Schmidt; M Vorgerd; M Winterholler; K Eger; S Zierz; M Deschauer
Journal:  J Inherit Metab Dis       Date:  2008-07-10       Impact factor: 4.982

2.  Glycogen storage disease type II: frequency of three common mutant alleles and their associated clinical phenotypes studied in 121 patients.

Authors:  M A Kroos; M Van der Kraan; O P Van Diggelen; W J Kleijer; A J Reuser; M J Van den Boogaard; M G Ausems; H K Ploos van Amstel; L Poenaru; M Nicolino
Journal:  J Med Genet       Date:  1995-10       Impact factor: 6.318

3.  c.1437G>A intron 9 substitution on acid α-glucosidase gene associated with classic infantile-onset Pompe disease phenotype.

Authors:  Andrés Morales; Mikaela I Poling; Marco T Páez; Julio Cabrera; Rodger J McCormick
Journal:  BMJ Case Rep       Date:  2015-07-09

4.  Identification of eight novel mutations of the acid alpha-glucosidase gene causing the infantile or juvenile form of glycogen storage disease type II.

Authors:  L Wan; C-C Lee; C-M Hsu; W-L Hwu; C-C Yang; C-H Tsai; F-J Tsai
Journal:  J Neurol       Date:  2008-05-06       Impact factor: 4.849

5.  Leaky splicing mutation in the acid maltase gene is associated with delayed onset of glycogenosis type II.

Authors:  C F Boerkoel; R Exelbert; C Nicastri; R C Nichols; F W Miller; P H Plotz; N Raben
Journal:  Am J Hum Genet       Date:  1995-04       Impact factor: 11.025

6.  Pompe disease diagnosis and management guideline.

Authors:  Priya S Kishnani; Robert D Steiner; Deeksha Bali; Kenneth Berger; Barry J Byrne; Laura E Case; Laura Case; John F Crowley; Steven Downs; R Rodney Howell; Richard M Kravitz; Joanne Mackey; Deborah Marsden; Anna Maria Martins; David S Millington; Marc Nicolino; Gwen O'Grady; Marc C Patterson; David M Rapoport; Alfred Slonim; Carolyn T Spencer; Cynthia J Tifft; Michael S Watson
Journal:  Genet Med       Date:  2006-05       Impact factor: 8.822

7.  A novel homozygous mutation at the GAA gene in Mexicans with early-onset Pompe disease.

Authors:  Carmen Esmer; Rosario Becerra-Becerra; Claudia Peña-Zepeda; Antonio Bravo-Oro
Journal:  Acta Myol       Date:  2013-10

8.  Clinical Laboratory Experience of Blood CRIM Testing in Infantile Pompe Disease.

Authors:  Deeksha S Bali; Jennifer L Goldstein; Catherine Rehder; Zoheb B Kazi; Kathryn L Berrier; Jian Dai; Priya S Kishnani
Journal:  Mol Genet Metab Rep       Date:  2015-12-01

9.  The infantile-onset form of Pompe disease: an autopsy diagnosis.

Authors:  Otávio César Cruz Dos Santos; Regina Schultz
Journal:  Autops Case Rep       Date:  2015-12-30

10.  A New Mutation Causing Severe Infantile-Onset Pompe Disease Responsive to Enzyme Replacement Therapy.

Authors:  Hossein Moravej; Anis Amirhakimi; Alireza Showraki; Hamid Amoozgar; Zahra Hadipour; Ghasem Nikfar
Journal:  Iran J Med Sci       Date:  2018-03
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