Literature DB >> 28649782

Heterozygous variants in ACTL6A, encoding a component of the BAF complex, are associated with intellectual disability.

Ronit Marom1, Mahim Jain1, Lindsay C Burrage1, I-Wen Song1, Brett H Graham1, Chester W Brown2, Servi J C Stevens3, Alexander P A Stegmann3, Andrew T Gunter4, Julie D Kaplan4, Ralitza H Gavrilova5,6, Marwan Shinawi7, Jill A Rosenfeld1, Yangjin Bae1, Alyssa A Tran1, Yuqing Chen1, James T Lu8, Richard A Gibbs1,9, Christine Eng1, Yaping Yang1, Justine Rousseau10, Bert B A de Vries11, Philippe M Campeau10, Brendan Lee1.   

Abstract

Pathogenic variants in genes encoding components of the BRG1-associated factor (BAF) chromatin remodeling complex have been associated with intellectual disability syndromes. We identified heterozygous, novel variants in ACTL6A, a gene encoding a component of the BAF complex, in three subjects with varying degrees of intellectual disability. Two subjects have missense variants affecting highly conserved amino acid residues within the actin-like domain. Missense mutations in the homologous region in yeast actin were previously reported to be dominant lethal and were associated with impaired binding of the human ACTL6A to β-actin and BRG1. A third subject has a splicing variant that creates an in-frame deletion. Our findings suggest that the variants identified in our subjects may have a deleterious effect on the function of the protein by disturbing the integrity of the BAF complex. Thus, ACTL6A gene mutation analysis should be considered in patients with intellectual disability, learning disabilities, or developmental language disorder.
© 2017 Wiley Periodicals, Inc.

Entities:  

Keywords:  ACTL6A; BAF complex; intellectual disability; speech delay

Mesh:

Substances:

Year:  2017        PMID: 28649782      PMCID: PMC5599325          DOI: 10.1002/humu.23282

Source DB:  PubMed          Journal:  Hum Mutat        ISSN: 1059-7794            Impact factor:   4.878


  45 in total

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Authors:  Veneta Krasteva; Manuel Buscarlet; Abigail Diaz-Tellez; Marie-Anne Bernard; Gerald R Crabtree; Julie A Lessard
Journal:  Blood       Date:  2012-09-26       Impact factor: 22.113

2.  Rapid and phosphoinositol-dependent binding of the SWI/SNF-like BAF complex to chromatin after T lymphocyte receptor signaling.

Authors:  K Zhao; W Wang; O J Rando; Y Xue; K Swiderek; A Kuo; G R Crabtree
Journal:  Cell       Date:  1998-11-25       Impact factor: 41.582

3.  DYRK1A haploinsufficiency causes a new recognizable syndrome with microcephaly, intellectual disability, speech impairment, and distinct facies.

Authors:  Jianling Ji; Hane Lee; Bob Argiropoulos; Naghmeh Dorrani; John Mann; Julian A Martinez-Agosto; Natalia Gomez-Ospina; Natalie Gallant; Jonathan A Bernstein; Louanne Hudgins; Leah Slattery; Bertrand Isidor; Cédric Le Caignec; Albert David; Ewa Obersztyn; Barbara Wiśniowiecka-Kowalnik; Michelle Fox; Joshua L Deignan; Eric Vilain; Emily Hendricks; Margaret Horton Harr; Sarah E Noon; Jessi R Jackson; Alisha Wilkens; Ghayda Mirzaa; Noriko Salamon; Jeff Abramson; Elaine H Zackai; Ian Krantz; A Micheil Innes; Stanley F Nelson; Wayne W Grody; Fabiola Quintero-Rivera
Journal:  Eur J Hum Genet       Date:  2015-05-06       Impact factor: 4.246

4.  Actl6a protects embryonic stem cells from differentiating into primitive endoderm.

Authors:  Weisi Lu; Lekun Fang; Bin Ouyang; Xiya Zhang; Shaoquan Zhan; Xuyang Feng; Yaofu Bai; Xin Han; Hyeung Kim; Quanyuan He; Ma Wan; Feng-Tao Shi; Xin-Hua Feng; Dan Liu; Junjiu Huang; Zhou Songyang
Journal:  Stem Cells       Date:  2015-06       Impact factor: 6.277

5.  GeneMatcher: a matching tool for connecting investigators with an interest in the same gene.

Authors:  Nara Sobreira; François Schiettecatte; David Valle; Ada Hamosh
Journal:  Hum Mutat       Date:  2015-08-13       Impact factor: 4.878

6.  Severe forms of Baraitser-Winter syndrome are caused by ACTB mutations rather than ACTG1 mutations.

Authors:  N Di Donato; A Rump; R Koenig; V M Der Kaloustian; F Halal; K Sonntag; C Krause; K Hackmann; G Hahn; E Schrock; A Verloes
Journal:  Eur J Hum Genet       Date:  2013-06-12       Impact factor: 4.246

7.  Heterocomplex formation by Arp4 and β-actin is involved in the integrity of the Brg1 chromatin remodeling complex.

Authors:  Naoki Nishimoto; Masanori Watanabe; Shinya Watanabe; Nozomi Sugimoto; Takashi Yugawa; Tsuyoshi Ikura; Osamu Koiwai; Tohru Kiyono; Masatoshi Fujita
Journal:  J Cell Sci       Date:  2012-05-08       Impact factor: 5.285

8.  I-TASSER server for protein 3D structure prediction.

Authors:  Yang Zhang
Journal:  BMC Bioinformatics       Date:  2008-01-23       Impact factor: 3.169

Review 9.  Role of nucleosome remodeling in neurodevelopmental and intellectual disability disorders.

Authors:  Alberto J López; Marcelo A Wood
Journal:  Front Behav Neurosci       Date:  2015-04-23       Impact factor: 3.558

10.  A global reference for human genetic variation.

Authors:  Adam Auton; Lisa D Brooks; Richard M Durbin; Erik P Garrison; Hyun Min Kang; Jan O Korbel; Jonathan L Marchini; Shane McCarthy; Gil A McVean; Gonçalo R Abecasis
Journal:  Nature       Date:  2015-10-01       Impact factor: 49.962

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  10 in total

1.  Expanding the Spectrum of BAF-Related Disorders: De Novo Variants in SMARCC2 Cause a Syndrome with Intellectual Disability and Developmental Delay.

Authors:  Keren Machol; Justine Rousseau; Sophie Ehresmann; Thomas Garcia; Thi Tuyet Mai Nguyen; Rebecca C Spillmann; Jennifer A Sullivan; Vandana Shashi; Yong-Hui Jiang; Nicholas Stong; Elise Fiala; Marcia Willing; Rolph Pfundt; Tjitske Kleefstra; Megan T Cho; Heather McLaughlin; Monica Rosello Piera; Carmen Orellana; Francisco Martínez; Alfonso Caro-Llopis; Sandra Monfort; Tony Roscioli; Cheng Yee Nixon; Michael F Buckley; Anne Turner; Wendy D Jones; Peter M van Hasselt; Floris C Hofstede; Koen L I van Gassen; Alice S Brooks; Marjon A van Slegtenhorst; Katherine Lachlan; Jessica Sebastian; Suneeta Madan-Khetarpal; Desai Sonal; Naidu Sakkubai; Julien Thevenon; Laurence Faivre; Alice Maurel; Slavé Petrovski; Ian D Krantz; Jennifer M Tarpinian; Jill A Rosenfeld; Brendan H Lee; Philippe M Campeau
Journal:  Am J Hum Genet       Date:  2018-12-20       Impact factor: 11.025

2.  Retrospective analysis of a clinical exome sequencing cohort reveals the mutational spectrum and identifies candidate disease-associated loci for BAFopathies.

Authors:  Chun-An Chen; John Lattier; Wenmiao Zhu; Jill Rosenfeld; Lei Wang; Tiana M Scott; Haowei Du; Vipulkumar Patel; Anh Dang; Pilar Magoulas; Haley Streff; Jessica Sebastian; Shayna Svihovec; Kathryn Curry; Mauricio R Delgado; Neil A Hanchard; Seema Lalani; Ronit Marom; Suneeta Madan-Khetarpal; Margarita Saenz; Hongzheng Dai; Linyan Meng; Fan Xia; Weimin Bi; Pengfei Liu; Jennifer E Posey; Daryl A Scott; James R Lupski; Christine M Eng; Rui Xiao; Bo Yuan
Journal:  Genet Med       Date:  2021-11-30       Impact factor: 8.822

3.  Modular Organization and Assembly of SWI/SNF Family Chromatin Remodeling Complexes.

Authors:  Nazar Mashtalir; Andrew R D'Avino; Brittany C Michel; Jie Luo; Joshua Pan; Jordan E Otto; Hayley J Zullow; Zachary M McKenzie; Rachel L Kubiak; Roodolph St Pierre; Alfredo M Valencia; Steven J Poynter; Seth H Cassel; Jeffrey A Ranish; Cigall Kadoch
Journal:  Cell       Date:  2018-10-18       Impact factor: 41.582

4.  A Syndromic Neurodevelopmental Disorder Caused by Mutations in SMARCD1, a Core SWI/SNF Subunit Needed for Context-Dependent Neuronal Gene Regulation in Flies.

Authors:  Kevin C J Nixon; Justine Rousseau; Max H Stone; Mohammed Sarikahya; Sophie Ehresmann; Seiji Mizuno; Naomichi Matsumoto; Noriko Miyake; Diana Baralle; Shane McKee; Kosuke Izumi; Alyssa L Ritter; Solveig Heide; Delphine Héron; Christel Depienne; Hannah Titheradge; Jamie M Kramer; Philippe M Campeau
Journal:  Am J Hum Genet       Date:  2019-03-14       Impact factor: 11.025

5.  Leveraging gene co-regulation to identify gene sets enriched for disease heritability.

Authors:  Katherine M Siewert-Rocks; Samuel S Kim; Douglas W Yao; Huwenbo Shi; Alkes L Price
Journal:  Am J Hum Genet       Date:  2022-02-01       Impact factor: 11.043

6.  De novo variant of TRRAP in a patient with very early onset psychosis in the context of non-verbal learning disability and obsessive-compulsive disorder: a case report.

Authors:  Chrystal F Mavros; Catherine A Brownstein; Roshni Thyagrajan; Casie A Genetti; Sahil Tembulkar; Kelsey Graber; Quinn Murphy; Kristin Cabral; Grace E VanNoy; Matthew Bainbridge; Jiahai Shi; Pankaj B Agrawal; Alan H Beggs; Eugene D'Angelo; Joseph Gonzalez-Heydrich
Journal:  BMC Med Genet       Date:  2018-11-13       Impact factor: 2.103

7.  ACTL6A knockdown inhibits cell migration by suppressing the AKT signaling pathway and enhances the sensitivity of glioma cells to temozolomide.

Authors:  Xueru Chen; Zijin Xiang; Dangchi Li; Xiuting Zhu; Xiangdong Peng
Journal:  Exp Ther Med       Date:  2020-12-28       Impact factor: 2.447

8.  Loss of the neural-specific BAF subunit ACTL6B relieves repression of early response genes and causes recessive autism.

Authors:  Wendy Wenderski; Lu Wang; Andrey Krokhotin; Jessica J Walsh; Hongjie Li; Hirotaka Shoji; Shereen Ghosh; Renee D George; Erik L Miller; Laura Elias; Mark A Gillespie; Esther Y Son; Brett T Staahl; Seung Tae Baek; Valentina Stanley; Cynthia Moncada; Zohar Shipony; Sara B Linker; Maria C N Marchetto; Fred H Gage; Dillon Chen; Tipu Sultan; Maha S Zaki; Jeffrey A Ranish; Tsuyoshi Miyakawa; Liqun Luo; Robert C Malenka; Gerald R Crabtree; Joseph G Gleeson
Journal:  Proc Natl Acad Sci U S A       Date:  2020-04-20       Impact factor: 12.779

9.  Intrinsic Disorder of the BAF Complex: Roles in Chromatin Remodeling and Disease Development.

Authors:  Nashwa El Hadidy; Vladimir N Uversky
Journal:  Int J Mol Sci       Date:  2019-10-23       Impact factor: 5.923

10.  GBAF, a small BAF sub-complex with big implications: a systematic review.

Authors:  Sarah M Innis; Birgit Cabot
Journal:  Epigenetics Chromatin       Date:  2020-11-03       Impact factor: 4.954

  10 in total

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