Literature DB >> 26407811

Late-Onset Combined Immunodeficiency with a Novel IL2RG Mutation and Probable Revertant Somatic Mosaicism.

Yusuke Okuno1, Akihiro Hoshino2, Hideki Muramatsu1, Nozomu Kawashima1, Xinan Wang1, Kenichi Yoshida3, Taizo Wada4, Masaharu Gunji5, Tomoko Toma4, Tamaki Kato6, Yuichi Shiraishi7, Atsuko Iwata8, Toshinori Hori8, Toshiyuki Kitoh8, Kenichi Chiba7, Hiroko Tanaka7, Masashi Sanada3, Yoshiyuki Takahashi1, Shigeaki Nonoyama6, Masafumi Ito5, Satoru Miyano7,9, Seishi Ogawa3, Seiji Kojima1, Hirokazu Kanegane10,11.   

Abstract

Primary immunodeficiency disease (PID) is caused by mutations of more than two hundred immunity-related genes. In addition to the heterogeneity of the diseases, the atypical presentation of each disease caused by hypomorphic mutations or somatic mosaicism makes genetic diagnosis challenging. Next-generation sequencing tests all genes simultaneously and has proven its innovative efficacy in genomics. We describe a male PID patient without any family history of immunodeficiency. This patient suffered from recurrent infections from 1 year of age. Laboratory analysis showed hypogammaglobulinemia. T, B, and NK cells were present, but the T cell proliferative response decreased. Whole-exome sequencing analysis identified an IL2RG p.P58T missense mutation. CD8(+) and CD56(+) cells showed revertant somatic mosaicism to the wild-type allele. A late-onset and atypical presentation of the X-linked severe combined immunodeficiency (X-SCID) phenotype might be associated with revertant somatic mosaicism in T and NK cells. This patient is the seventh reported case of X-SCID with revertant somatic mosaicism. His classical clinical management did not result in a molecular diagnosis because of the atypical presentation. The coverage that is provided by whole-exome sequencing of most PID genes effectively excluded differential diagnoses other than X-SCID. As next-generation sequencing becomes available in clinical practice, it will enhance our knowledge of PID and rescue currently undiagnosed patients.

Entities:  

Keywords:  Combined immunodeficiency; Revertant somatic mosaicism; Whole-exome sequencing; X-linked severe combined immunodeficiency

Mesh:

Substances:

Year:  2015        PMID: 26407811     DOI: 10.1007/s10875-015-0202-0

Source DB:  PubMed          Journal:  J Clin Immunol        ISSN: 0271-9142            Impact factor:   8.317


  12 in total

1.  Atypical X-linked severe combined immunodeficiency due to possible spontaneous reversion of the genetic defect in T cells.

Authors:  V Stephan; V Wahn; F Le Deist; U Dirksen; B Broker; I Müller-Fleckenstein; G Horneff; H Schroten; A Fischer; G de Saint Basile
Journal:  N Engl J Med       Date:  1996-11-21       Impact factor: 91.245

2.  ACTN1 mutations cause congenital macrothrombocytopenia.

Authors:  Shinji Kunishima; Yusuke Okuno; Kenichi Yoshida; Yuichi Shiraishi; Masashi Sanada; Hideki Muramatsu; Kenichi Chiba; Hiroko Tanaka; Koji Miyazaki; Michio Sakai; Masatoshi Ohtake; Ryoji Kobayashi; Akihiro Iguchi; Gen Niimi; Makoto Otsu; Yoshiyuki Takahashi; Satoru Miyano; Hidehiko Saito; Seiji Kojima; Seishi Ogawa
Journal:  Am J Hum Genet       Date:  2013-02-21       Impact factor: 11.025

3.  IL2RG reversion event in a common lymphoid progenitor leads to delayed diagnosis and milder phenotype.

Authors:  Amy P Hsu; Stefania Pittaluga; Bianca Martinez; Amy P Rump; Mark Raffeld; Gulbu Uzel; Jennifer M Puck; Alexandra F Freeman; Steven M Holland
Journal:  J Clin Immunol       Date:  2015-06-17       Impact factor: 8.317

4.  Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States.

Authors:  Antonia Kwan; Roshini S Abraham; Robert Currier; Amy Brower; Karen Andruszewski; Jordan K Abbott; Mei Baker; Mark Ballow; Louis E Bartoshesky; Francisco A Bonilla; Charles Brokopp; Edward Brooks; Michele Caggana; Jocelyn Celestin; Joseph A Church; Anne Marie Comeau; James A Connelly; Morton J Cowan; Charlotte Cunningham-Rundles; Trivikram Dasu; Nina Dave; Maria T De La Morena; Ulrich Duffner; Chin-To Fong; Lisa Forbes; Debra Freedenberg; Erwin W Gelfand; Jaime E Hale; I Celine Hanson; Beverly N Hay; Diana Hu; Anthony Infante; Daisy Johnson; Neena Kapoor; Denise M Kay; Donald B Kohn; Rachel Lee; Heather Lehman; Zhili Lin; Fred Lorey; Aly Abdel-Mageed; Adrienne Manning; Sean McGhee; Theodore B Moore; Stanley J Naides; Luigi D Notarangelo; Jordan S Orange; Sung-Yun Pai; Matthew Porteus; Ray Rodriguez; Neil Romberg; John Routes; Mary Ruehle; Arye Rubenstein; Carlos A Saavedra-Matiz; Ginger Scott; Patricia M Scott; Elizabeth Secord; Christine Seroogy; William T Shearer; Subhadra Siegel; Stacy K Silvers; E Richard Stiehm; Robert W Sugerman; John L Sullivan; Susan Tanksley; Millard L Tierce; James Verbsky; Beth Vogel; Rosalyn Walker; Kelly Walkovich; Jolan E Walter; Richard L Wasserman; Michael S Watson; Geoffrey A Weinberg; Leonard B Weiner; Heather Wood; Anne B Yates; Jennifer M Puck; Vincent R Bonagura
Journal:  JAMA       Date:  2014-08-20       Impact factor: 56.272

5.  Multiple reversions of an IL2RG mutation restore T cell function in an X-linked severe combined immunodeficiency patient.

Authors:  Tomoki Kawai; Megumu Saito; Ryuta Nishikomori; Takahiro Yasumi; Kazushi Izawa; Tomohiko Murakami; Shigefumi Okamoto; Yasuko Mori; Noriko Nakagawa; Kohsuke Imai; Shigeaki Nonoyama; Taizo Wada; Akihiro Yachie; Katsuyuki Ohmori; Tatsutoshi Nakahata; Toshio Heike
Journal:  J Clin Immunol       Date:  2012-03-30       Impact factor: 8.317

6.  Frequent pathway mutations of splicing machinery in myelodysplasia.

Authors:  Kenichi Yoshida; Masashi Sanada; Yuichi Shiraishi; Daniel Nowak; Yasunobu Nagata; Ryo Yamamoto; Yusuke Sato; Aiko Sato-Otsubo; Ayana Kon; Masao Nagasaki; George Chalkidis; Yutaka Suzuki; Masashi Shiosaka; Ryoichiro Kawahata; Tomoyuki Yamaguchi; Makoto Otsu; Naoshi Obara; Mamiko Sakata-Yanagimoto; Ken Ishiyama; Hiraku Mori; Florian Nolte; Wolf-Karsten Hofmann; Shuichi Miyawaki; Sumio Sugano; Claudia Haferlach; H Phillip Koeffler; Lee-Yung Shih; Torsten Haferlach; Shigeru Chiba; Hiromitsu Nakauchi; Satoru Miyano; Seishi Ogawa
Journal:  Nature       Date:  2011-09-11       Impact factor: 49.962

7.  A reversion of an IL2RG mutation in combined immunodeficiency providing competitive advantage to the majority of CD8+ T cells.

Authors:  Taco W Kuijpers; Ester M M van Leeuwen; Barbara H Barendregt; Paul Klarenbeek; Daan J aan de Kerk; Paul A Baars; Machiel H Jansen; Niek de Vries; René A W van Lier; Mirjam van der Burg
Journal:  Haematologica       Date:  2013-02-12       Impact factor: 9.941

8.  Detection of T lymphocytes with a second-site mutation in skin lesions of atypical X-linked severe combined immunodeficiency mimicking Omenn syndrome.

Authors:  Taizo Wada; Masahiro Yasui; Tomoko Toma; Yuko Nakayama; Mika Nishida; Masaki Shimizu; Michiko Okajima; Yoshihito Kasahara; Shoichi Koizumi; Masami Inoue; Keisei Kawa; Akihiro Yachie
Journal:  Blood       Date:  2008-06-16       Impact factor: 22.113

9.  Primary Immune Deficiency Treatment Consortium (PIDTC) report.

Authors:  Linda M Griffith; Morton J Cowan; Luigi D Notarangelo; Donald B Kohn; Jennifer M Puck; Sung-Yun Pai; Barbara Ballard; Sarah C Bauer; Jack J H Bleesing; Marcia Boyle; Amy Brower; Rebecca H Buckley; Mirjam van der Burg; Lauri M Burroughs; Fabio Candotti; Andrew J Cant; Talal Chatila; Charlotte Cunningham-Rundles; Mary C Dinauer; Christopher C Dvorak; Alexandra H Filipovich; Thomas A Fleisher; Hubert Bobby Gaspar; Tayfun Gungor; Elie Haddad; Emily Hovermale; Faith Huang; Alan Hurley; Mary Hurley; Sumathi Iyengar; Elizabeth M Kang; Brent R Logan; Janel R Long-Boyle; Harry L Malech; Sean A McGhee; Fred Modell; Vicki Modell; Hans D Ochs; Richard J O'Reilly; Robertson Parkman; David J Rawlings; John M Routes; William T Shearer; Trudy N Small; Heather Smith; Kathleen E Sullivan; Paul Szabolcs; Adrian Thrasher; Troy R Torgerson; Paul Veys; Kenneth Weinberg; Juan Carlos Zuniga-Pflucker
Journal:  J Allergy Clin Immunol       Date:  2013-10-15       Impact factor: 10.793

10.  A novel immunodeficiency associated with hypomorphic RAG1 mutations and CMV infection.

Authors:  Jean-Pierre de Villartay; Annick Lim; Hamoud Al-Mousa; Sophie Dupont; Julie Déchanet-Merville; Edith Coumau-Gatbois; Marie-Lise Gougeon; Arnaud Lemainque; Céline Eidenschenk; Emmanuelle Jouanguy; Laurent Abel; Jean-Laurent Casanova; Alain Fischer; Françoise Le Deist
Journal:  J Clin Invest       Date:  2005-11       Impact factor: 14.808

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  11 in total

1.  A synonymous splice site mutation in IL2RG gene causes late-onset combined immunodeficiency.

Authors:  Motoi Yamashita; Ryosuke Wakatsuki; Tamaki Kato; Tsubasa Okano; Shingo Yamanishi; Nobuko Mayumi; Mayuri Tanaka; Yumi Ogura; Hirokazu Kanegane; Shigeaki Nonoyama; Kohsuke Imai; Tomohiro Morio
Journal:  Int J Hematol       Date:  2019-03-08       Impact factor: 2.490

2.  Treatment of Relapsing HPV Diseases by Restored Function of Natural Killer Cells.

Authors:  Andrea Lisco; Amy P Hsu; Dimana Dimitrova; Diana M Proctor; Emily M Mace; Peiying Ye; Megan V Anderson; Stephanie N Hicks; Christopher Grivas; Dima A Hammoud; Maura Manion; Gabriel J Starrett; Alvin Farrel; Kerry Dobbs; Isaac Brownell; Christopher Buck; Luigi D Notarangelo; Jordan S Orange; Warren J Leonard; Michael I Orestes; Anju T Peters; Jennifer A Kanakry; Julia A Segre; Heidi H Kong; Irini Sereti
Journal:  N Engl J Med       Date:  2021-09-02       Impact factor: 176.079

3.  The IL-2RG R328X nonsense mutation allows partial STAT-5 phosphorylation and defines a critical region involved in the leaky-SCID phenotype.

Authors:  A Arcas-García; M Garcia-Prat; M Magallón-Lorenz; A Martín-Nalda; O Drechsel; S Ossowski; L Alonso; J G Rivière; P Soler-Palacín; R Colobran; J Sayós; M Martínez-Gallo; C Franco-Jarava
Journal:  Clin Exp Immunol       Date:  2020-01-19       Impact factor: 4.330

4.  A Novel de Novo Mutation in the CD40 Ligand Gene in a Patient With a Mild X-Linked Hyper-IgM Phenotype Initially Diagnosed as CVID: New Aspects of Old Diseases.

Authors:  Tábata T França; Luiz F B Leite; Tiago A Maximo; Christiane G Lambert; Nuria B Zurro; Wilma C N Forte; Antonio Condino-Neto
Journal:  Front Pediatr       Date:  2018-05-04       Impact factor: 3.418

5.  Clinical, Immunological, and Molecular Features of Typical and Atypical Severe Combined Immunodeficiency: Report of the Italian Primary Immunodeficiency Network.

Authors:  Emilia Cirillo; Caterina Cancrini; Chiara Azzari; Silvana Martino; Baldassarre Martire; Andrea Pession; Alberto Tommasini; Samuele Naviglio; Andrea Finocchi; Rita Consolini; Paolo Pierani; Irene D'Alba; Maria Caterina Putti; Antonio Marzollo; Giuliana Giardino; Rosaria Prencipe; Federica Esposito; Fiorentino Grasso; Alessia Scarselli; Gigliola Di Matteo; Enrico Attardi; Silvia Ricci; Davide Montin; Fernando Specchia; Federica Barzaghi; Maria Pia Cicalese; Giuseppe Quaremba; Vassilios Lougaris; Silvia Giliani; Franco Locatelli; Paolo Rossi; Alessandro Aiuti; Raffaele Badolato; Alessandro Plebani; Claudio Pignata
Journal:  Front Immunol       Date:  2019-08-13       Impact factor: 7.561

6.  Novel Hemizygous IL2RG p.(Pro58Ser) Mutation Impairs IL-2 Receptor Complex Expression on Lymphocytes Causing X-Linked Combined Immunodeficiency.

Authors:  Elina A Tuovinen; Juha Grönholm; Tiina Öhman; Sakari Pöysti; Raine Toivonen; Anna Kreutzman; Kaarina Heiskanen; Luca Trotta; Sanna Toiviainen-Salo; John M Routes; James Verbsky; Satu Mustjoki; Janna Saarela; Juha Kere; Markku Varjosalo; Arno Hänninen; Mikko R J Seppänen
Journal:  J Clin Immunol       Date:  2020-02-19       Impact factor: 8.317

Review 7.  Genetic Mosaicism as a Cause of Inborn Errors of Immunity.

Authors:  Jahnavi Aluri; Megan A Cooper
Journal:  J Clin Immunol       Date:  2021-04-16       Impact factor: 8.317

Review 8.  Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.

Authors:  Elena Blanco; Natalia Izotova; Claire Booth; Adrian James Thrasher
Journal:  Front Immunol       Date:  2020-11-27       Impact factor: 7.561

9.  Case Report: Interleukin-2 Receptor Common Gamma Chain Defect Presented as a Hyper-IgE Syndrome.

Authors:  Brahim Belaid; Lydia Lamara Mahammed; Aida Mohand Oussaid; Melanie Migaud; Yasmine Khadri; Jean Laurent Casanova; Anne Puel; Nafissa Ben Halla; Reda Djidjik
Journal:  Front Immunol       Date:  2021-06-24       Impact factor: 7.561

10.  Somatic Reversion of a Novel IL2RG Mutation Resulting in Atypical X-Linked Combined Immunodeficiency.

Authors:  Yujuan Hou; Hans Peter Gratz; Guillermo Ureña-Bailén; Paul G Gratz; Karin Schilbach-Stückle; Tina Renno; Derya Güngör; Daniel A Mader; Elke Malenke; Justin S Antony; Rupert Handgretinger; Markus Mezger
Journal:  Genes (Basel)       Date:  2021-12-23       Impact factor: 4.096

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