Literature DB >> 19734074

Type 2 Gaucher disease occurs in Ashkenazi Jews but is surprisingly rare.

Shraga Aviner1, Ben-Zion Garty, Avinoam Rachmel, Hagit N Baris, Ellen Sidransky, Avinoam Shuffer, Joseph Attias, Yisaac Yaniv, Ian J Cohen.   

Abstract

Patients with Gaucher disease (GD) are divided into three types based on the presence and rate of progression of the neurologic manifestations. While type 1 GD has a strong predilection in the Jewish Ashkenazi population, both other types lack such a propensity. We report the occurrence of type 2 GD (GD2) in four pregnancies in two Jewish families in Israel (in one case the mother was not Ashkenazi but was from a Sfaradi Jewish family) and also review seven additional cases of GD2 in Ashkenazi Jewish families reported in the literature. Phenotypically, GD2 in Ashkenazi Jews does not differ significantly from this form in other ethnic groups. Genotypic analysis of probands from the two Israeli families demonstrates that each carried two heterozygous glucocerebrosidase mutations. We could find no explanation why GD2 is so rare in the Jewish Ashkenazi population but we could hypothesize that homozygosity for certain Ashkenazi alleles might be lethal, leading to a lower than expected frequency of GD2 and noted that no cases of homozygous L444P has ever been described in Ashkenazi Jews.

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Year:  2009        PMID: 19734074      PMCID: PMC3355376          DOI: 10.1016/j.bcmd.2009.08.004

Source DB:  PubMed          Journal:  Blood Cells Mol Dis        ISSN: 1079-9796            Impact factor:   3.039


  11 in total

Review 1.  Type 2 gaucher disease: an expanding phenotype.

Authors:  N Tayebi; D L Stone; E Sidransky
Journal:  Mol Genet Metab       Date:  1999-10       Impact factor: 4.797

2.  [Not Available].

Authors:  E SCHAIRER
Journal:  Virchows Arch Pathol Anat Physiol Klin Med       Date:  1948

Review 3.  Glucocerebrosidase gene mutations in patients with type 2 Gaucher disease.

Authors:  D L Stone; N Tayebi; E Orvisky; B Stubblefield; V Madike; E Sidransky
Journal:  Hum Mutat       Date:  2000       Impact factor: 4.878

4.  Gaucher disease--Norrbottnian type. Neurodevelopmental, neurological, and neurophysiological aspects.

Authors:  S Blom; A Erikson
Journal:  Eur J Pediatr       Date:  1983-09       Impact factor: 3.183

5.  Fine structure of central nervous system in early infantile Gaucher's disease.

Authors:  M Adachi; B J Wallace; L Schneck; B W Volk
Journal:  Arch Pathol       Date:  1967-06

6.  Divergent phenotypes in Gaucher disease implicate the role of modifiers.

Authors:  O Goker-Alpan; K S Hruska; E Orvisky; P S Kishnani; B K Stubblefield; R Schiffmann; E Sidransky
Journal:  J Med Genet       Date:  2005-06       Impact factor: 6.318

7.  Analysis and classification of 304 mutant alleles in patients with type 1 and type 3 Gaucher disease.

Authors:  V Koprivica; D L Stone; J K Park; M Callahan; A Frisch; I J Cohen; N Tayebi; E Sidransky
Journal:  Am J Hum Genet       Date:  2000-05-04       Impact factor: 11.025

8.  Glucosylsphingosine accumulation in mice and patients with type 2 Gaucher disease begins early in gestation.

Authors:  E Orvisky; E Sidransky; C E McKinney; M E Lamarca; R Samimi; D Krasnewich; B M Martin; E I Ginns
Journal:  Pediatr Res       Date:  2000-08       Impact factor: 3.756

Review 9.  Type 2 Gaucher disease: 15 new cases and review of the literature.

Authors:  Cyril Mignot; Diana Doummar; Irène Maire; Thierry Billette De Villemeur
Journal:  Brain Dev       Date:  2006-01       Impact factor: 1.961

10.  Type 2 Gaucher disease with hydrops fetalis in an Ashkenazi Jewish family resulting from a novel recombinant allele and a rare splice junction mutation in the glucocerebrosidase locus.

Authors:  K Reissner; N Tayebi; B K Stubblefield; V Koprivica; M Blitzer; W Holleran; T Cowan; S Almashanu; A Maddalena; E M Karson; E Sidransky
Journal:  Mol Genet Metab       Date:  1998-04       Impact factor: 4.797

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  4 in total

Review 1.  The clinical management of Type 2 Gaucher disease.

Authors:  Karin Weiss; Ashley Gonzalez; Grisel Lopez; Leah Pedoeim; Catherine Groden; Ellen Sidransky
Journal:  Mol Genet Metab       Date:  2014-11-14       Impact factor: 4.797

2.  A germline or de novo mutation in two families with Gaucher disease: implications for recessive disorders.

Authors:  Hamid Saranjam; Sameer S Chopra; Harvey Levy; Barbara K Stubblefield; Emerson Maniwang; Ian J Cohen; Hagit Baris; Ellen Sidransky; Nahid Tayebi
Journal:  Eur J Hum Genet       Date:  2012-06-20       Impact factor: 4.246

Review 3.  Type 2 Gaucher disease: phenotypic variation and genotypic heterogeneity.

Authors:  N Gupta; I M Oppenheim; E F Kauvar; N Tayebi; E Sidransky
Journal:  Blood Cells Mol Dis       Date:  2010-09-28       Impact factor: 3.039

4.  Glucosylsphingosine is a highly sensitive and specific biomarker for primary diagnostic and follow-up monitoring in Gaucher disease in a non-Jewish, Caucasian cohort of Gaucher disease patients.

Authors:  Arndt Rolfs; Anne-Katrin Giese; Ulrike Grittner; Daniel Mascher; Deborah Elstein; Ari Zimran; Tobias Böttcher; Jan Lukas; Rayk Hübner; Uta Gölnitz; Anja Röhle; Ales Dudesek; Wolfgang Meyer; Matthias Wittstock; Hermann Mascher
Journal:  PLoS One       Date:  2013-11-20       Impact factor: 3.240

  4 in total

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