Literature DB >> 12743242

Hereditary inclusion body myopathy: the Middle Eastern genetic cluster.

Z Argov1, I Eisenberg, G Grabov-Nardini, M Sadeh, I Wirguin, D Soffer, S Mitrani-Rosenbaum.   

Abstract

BACKGROUND: Recessively inherited hereditary inclusion body myopathy (HIBM) with quadriceps sparing was initially described only in Jews originating from the region of Persia. The recent identification of the gene responsible for this myopathy and the common "Persian Jewish mutation" (M712T) enabled the re-evaluation of atypical phenotypes and the epidemiology of HIBM in various communities in the Middle East.
OBJECTIVE: To test for the M712T mutation in the DNA from HIBM patients in the Middle East.
METHODS: DNA from all suspected HIBM patients was tested for the M712T mutation. Unaffected members of families with genetically proven HIBM were studied too. In the majority of families, haplotype construction with markers spanning the 700-kb region of the HIBM gene was performed.
RESULTS: One hundred twenty-nine HIBM patients of 55 families (Middle Eastern Jews, Karaites, and Arab Muslims of Palestinian and Bedouin origin) were homozygous for the M712T mutation, and all carried the same haplotype. Five clinically unaffected subjects were also homozygous for the common mutation and haplotype, including two older adults (ages 50 and 68 years). Atypical features with this same mutation were marked quadriceps weakness in five patients, proximal weakness only in two patients, facial weakness in three patients, and a muscle biopsy showing perivascular inflammation in one patient.
CONCLUSIONS: The phenotypic spectrum of recessive HIBM is wider than previously described, and the diagnostic criteria for this myopathy must be changed. The Middle Eastern cluster is the result of a founder mutation, with incomplete penetrance, that is approximately 1,300 years old and is not limited to Jews.

Entities:  

Mesh:

Substances:

Year:  2003        PMID: 12743242     DOI: 10.1212/01.wnl.0000061617.71839.42

Source DB:  PubMed          Journal:  Neurology        ISSN: 0028-3878            Impact factor:   9.910


  31 in total

1.  Patient reported outcomes in GNE myopathy: incorporating a valid assessment of physical function in a rare disease.

Authors:  Christina Slota; Margaret Bevans; Li Yang; Joseph Shrader; Galen Joe; Nuria Carrillo
Journal:  Disabil Rehabil       Date:  2017-02-07       Impact factor: 3.033

Review 2.  Mutation update for GNE gene variants associated with GNE myopathy.

Authors:  Frank V Celeste; Thierry Vilboux; Carla Ciccone; John Karl de Dios; May Christine V Malicdan; Petcharat Leoyklang; John C McKew; William A Gahl; Nuria Carrillo-Carrasco; Marjan Huizing
Journal:  Hum Mutat       Date:  2014-08       Impact factor: 4.878

3.  Genetics of GNE myopathy in the non-Jewish Persian population.

Authors:  Alireza Haghighi; Shahriar Nafissi; Abrar Qurashi; Zheng Tan; Hosein Shamshiri; Yalda Nilipour; Amirreza Haghighi; Robert J Desnick; Ruth Kornreich
Journal:  Eur J Hum Genet       Date:  2015-05-13       Impact factor: 4.246

4.  Novel GNE mutations in two phenotypically distinct HIBM2 patients.

Authors:  Conrad C Weihl; Sara E Miller; Craig M Zaidman; Alan Pestronk; Robert H Baloh; Mohammed Al-Lozi
Journal:  Neuromuscul Disord       Date:  2010-12-04       Impact factor: 4.296

5.  Muscle imaging findings in GNE myopathy.

Authors:  Giorgio Tasca; Enzo Ricci; Mauro Monforte; Francesco Laschena; Pierfrancesco Ottaviani; Carmelo Rodolico; Emanuele Barca; Gabriella Silvestri; Elisabetta Iannaccone; Massimiliano Mirabella; Aldobrando Broccolini
Journal:  J Neurol       Date:  2012-01-10       Impact factor: 4.849

Review 6.  Distal myopathies.

Authors:  Mazen M Dimachkie; Richard J Barohn
Journal:  Neurol Clin       Date:  2014-05-15       Impact factor: 3.806

Review 7.  GNE myopathy: current update and future therapy.

Authors:  Ichizo Nishino; Nuria Carrillo-Carrasco; Zohar Argov
Journal:  J Neurol Neurosurg Psychiatry       Date:  2014-07-07       Impact factor: 10.154

8.  The Interaction of UDP-N-Acetylglucosamine 2-Epimerase/N-Acetylmannosamine Kinase (GNE) and Alpha-Actinin 2 Is Altered in GNE Myopathy M743T Mutant.

Authors:  Avi Harazi; Michal Becker-Cohen; Hagit Zer; Ofra Moshel; Stephan Hinderlich; Stella Mitrani-Rosenbaum
Journal:  Mol Neurobiol       Date:  2016-03-29       Impact factor: 5.590

9.  Hereditary Inclusion Body Myopathy (HIBM2).

Authors:  Chris M Jay; Nick Levonyak; Gregory Nemunaitis; Phillip B Maples; John Nemunaitis
Journal:  Gene Regul Syst Bio       Date:  2009-10-21

10.  Preclinical assessment of wt GNE gene plasmid for management of hereditary inclusion body myopathy 2 (HIBM2).

Authors:  Chris Jay; Gregory Nemunaitis; John Nemunaitis; Neil Senzer; Stephan Hinderlich; Daniel Darvish; Julie Ogden; John Eager; Alex Tong; Phillip B Maples
Journal:  Gene Regul Syst Bio       Date:  2008-06-20
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.