Literature DB >> 22323755

CAG repeat expansion in Huntington disease determines age at onset in a fully dominant fashion.

J-M Lee1, E M Ramos, J-H Lee, T Gillis, J S Mysore, M R Hayden, S C Warby, P Morrison, M Nance, C A Ross, R L Margolis, F Squitieri, S Orobello, S Di Donato, E Gomez-Tortosa, C Ayuso, O Suchowersky, R J A Trent, E McCusker, A Novelletto, M Frontali, R Jones, T Ashizawa, S Frank, M H Saint-Hilaire, S M Hersch, H D Rosas, D Lucente, M B Harrison, A Zanko, R K Abramson, K Marder, J Sequeiros, J S Paulsen, G B Landwehrmeyer, R H Myers, M E MacDonald, J F Gusella.   

Abstract

OBJECTIVE: Age at onset of diagnostic motor manifestations in Huntington disease (HD) is strongly correlated with an expanded CAG trinucleotide repeat. The length of the normal CAG repeat allele has been reported also to influence age at onset, in interaction with the expanded allele. Due to profound implications for disease mechanism and modification, we tested whether the normal allele, interaction between the expanded and normal alleles, or presence of a second expanded allele affects age at onset of HD motor signs.
METHODS: We modeled natural log-transformed age at onset as a function of CAG repeat lengths of expanded and normal alleles and their interaction by linear regression.
RESULTS: An apparently significant effect of interaction on age at motor onset among 4,068 subjects was dependent on a single outlier data point. A rigorous statistical analysis with a well-behaved dataset that conformed to the fundamental assumptions of linear regression (e.g., constant variance and normally distributed error) revealed significance only for the expanded CAG repeat, with no effect of the normal CAG repeat. Ten subjects with 2 expanded alleles showed an age at motor onset consistent with the length of the larger expanded allele.
CONCLUSIONS: Normal allele CAG length, interaction between expanded and normal alleles, and presence of a second expanded allele do not influence age at onset of motor manifestations, indicating that the rate of HD pathogenesis leading to motor diagnosis is determined by a completely dominant action of the longest expanded allele and as yet unidentified genetic or environmental factors.

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Year:  2012        PMID: 22323755      PMCID: PMC3306163          DOI: 10.1212/WNL.0b013e318249f683

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


  21 in total

1.  A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group.

Authors: 
Journal:  Cell       Date:  1993-03-26       Impact factor: 41.582

2.  A worldwide study of the Huntington's disease mutation. The sensitivity and specificity of measuring CAG repeats.

Authors:  B Kremer; P Goldberg; S E Andrew; J Theilmann; H Telenius; J Zeisler; F Squitieri; B Lin; A Bassett; E Almqvist
Journal:  N Engl J Med       Date:  1994-05-19       Impact factor: 91.245

3.  Homozygote for Huntington disease.

Authors:  R H Myers; J Leavitt; L A Farrer; J Jagadeesh; H McFarlane; C A Mastromauro; R J Mark; J F Gusella
Journal:  Am J Hum Genet       Date:  1989-10       Impact factor: 11.025

4.  The number of CAG repeats within the normal allele does not influence the age of onset in Huntington's disease.

Authors:  Jiří Klempíř; Jana Zidovská; Jan Stochl; Věra Kebrdlová Ing; Tereza Uhrová; Jan Roth
Journal:  Mov Disord       Date:  2010-11-10       Impact factor: 10.338

5.  DNA analysis of Huntington's disease: five years of experience in Germany, Austria, and Switzerland.

Authors:  F Laccone; U Engel; E Holinski-Feder; M Weigell-Weber; K Marczinek; D Nolte; D J Morris-Rosendahl; C Zühlke; K Fuchs; H Weirich-Schwaiger; G Schlüter; G von Beust; A M Vieira-Saecker; B H Weber; O Riess
Journal:  Neurology       Date:  1999-09-11       Impact factor: 9.910

6.  The relationship between trinucleotide (CAG) repeat length and clinical features of Huntington's disease.

Authors:  S E Andrew; Y P Goldberg; B Kremer; H Telenius; J Theilmann; S Adam; E Starr; F Squitieri; B Lin; M A Kalchman
Journal:  Nat Genet       Date:  1993-08       Impact factor: 38.330

7.  Trinucleotide repeat length instability and age of onset in Huntington's disease.

Authors:  M Duyao; C Ambrose; R Myers; A Novelletto; F Persichetti; M Frontali; S Folstein; C Ross; M Franz; M Abbott
Journal:  Nat Genet       Date:  1993-08       Impact factor: 38.330

8.  A new polymerase chain reaction (PCR) assay for the trinucleotide repeat that is unstable and expanded on Huntington's disease chromosomes.

Authors:  J P Warner; L H Barron; D J Brock
Journal:  Mol Cell Probes       Date:  1993-06       Impact factor: 2.365

9.  The normal Huntington disease (HD) allele, or a closely linked gene, influences age at onset of HD.

Authors:  L A Farrer; L A Cupples; P Wiater; P M Conneally; J F Gusella; R H Myers
Journal:  Am J Hum Genet       Date:  1993-07       Impact factor: 11.025

10.  A genome scan for modifiers of age at onset in Huntington disease: The HD MAPS study.

Authors:  Jian-Liang Li; Michael R Hayden; Elisabeth W Almqvist; Ryan R Brinkman; Alexandra Durr; Catherine Dodé; Patrick J Morrison; Oksana Suchowersky; Christopher A Ross; Russell L Margolis; Adam Rosenblatt; Estrella Gómez-Tortosa; David Mayo Cabrero; Andrea Novelletto; Marina Frontali; Martha Nance; Ronald J A Trent; Elizabeth McCusker; Randi Jones; Jane S Paulsen; Madeline Harrison; Andrea Zanko; Ruth K Abramson; Ana L Russ; Beth Knowlton; Luc Djoussé; Jayalakshmi S Mysore; Suzanne Tariot; Michael F Gusella; Vanessa C Wheeler; Larry D Atwood; L Adrienne Cupples; Marie Saint-Hilaire; Jang-Ho J Cha; Steven M Hersch; Walter J Koroshetz; James F Gusella; Marcy E MacDonald; Richard H Myers
Journal:  Am J Hum Genet       Date:  2003-08-01       Impact factor: 11.025

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  139 in total

Review 1.  Huntington Disease: Linking Pathogenesis to the Development of Experimental Therapeutics.

Authors:  Tiago A Mestre; Cristina Sampaio
Journal:  Curr Neurol Neurosci Rep       Date:  2017-02       Impact factor: 5.081

2.  Cell biology. A unifying role for prions in neurodegenerative diseases.

Authors:  Stanley B Prusiner
Journal:  Science       Date:  2012-06-22       Impact factor: 47.728

3.  TAA repeat variation in the GRIK2 gene does not influence age at onset in Huntington's disease.

Authors:  Ji-Hyun Lee; Jong-Min Lee; Eliana Marisa Ramos; Tammy Gillis; Jayalakshmi S Mysore; Shotaro Kishikawa; Tiffany Hadzi; Audrey E Hendricks; Michael R Hayden; Patrick J Morrison; Martha Nance; Christopher A Ross; Russell L Margolis; Ferdinando Squitieri; Cinzia Gellera; Estrella Gomez-Tortosa; Carmen Ayuso; Oksana Suchowersky; Ronald J Trent; Elizabeth McCusker; Andrea Novelletto; Marina Frontali; Randi Jones; Tetsuo Ashizawa; Samuel Frank; Marie-Helene Saint-Hilaire; Steven M Hersch; Herminia D Rosas; Diane Lucente; Madaline B Harrison; Andrea Zanko; Ruth K Abramson; Karen Marder; Jorge Sequeiros; G Bernhard Landwehrmeyer; Ira Shoulson; Richard H Myers; Marcy E MacDonald; James F Gusella
Journal:  Biochem Biophys Res Commun       Date:  2012-07-03       Impact factor: 3.575

4.  Nonparametric modeling and analysis of association between Huntington's disease onset and CAG repeats.

Authors:  Yanyuan Ma; Yuanjia Wang
Journal:  Stat Med       Date:  2013-09-12       Impact factor: 2.373

Review 5.  Clinical Neurology and Epidemiology of the Major Neurodegenerative Diseases.

Authors:  Michael G Erkkinen; Mee-Ohk Kim; Michael D Geschwind
Journal:  Cold Spring Harb Perspect Biol       Date:  2018-04-02       Impact factor: 10.005

6.  Tracking motor impairments in the progression of Huntington's disease.

Authors:  Jeffery D Long; Jane S Paulsen; Karen Marder; Ying Zhang; Ji-In Kim; James A Mills
Journal:  Mov Disord       Date:  2013-10-21       Impact factor: 10.338

Review 7.  Progress and prospects for genetic modification of nonhuman primate models in biomedical research.

Authors:  Anthony W S Chan
Journal:  ILAR J       Date:  2013

Review 8.  Proteostasis in Huntington's disease: disease mechanisms and therapeutic opportunities.

Authors:  Rachel J Harding; Yu-Feng Tong
Journal:  Acta Pharmacol Sin       Date:  2018-04-05       Impact factor: 6.150

9.  Association of age at onset in Huntington disease with functional promoter variations in NPY and NPY2R.

Authors:  Eugen Kloster; Carsten Saft; Denis A Akkad; Jörg T Epplen; Larissa Arning
Journal:  J Mol Med (Berl)       Date:  2014-02       Impact factor: 4.599

Review 10.  Next-generation sequencing diagnostics for neurological diseases/disorders: from a clinical perspective.

Authors:  Jia Nee Foo; Jianjun Liu; Eng-King Tan
Journal:  Hum Genet       Date:  2013-03-23       Impact factor: 4.132

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