Clinical and Genetic Investigation of a Japanese Family With Cardiac Fabry Disease Identification of a Novel .ALPHA.-Galactosidase A Missense Mutation (G195V)

  • Seino Utako
    Department of Clinical Nephroscience, Niigata University Graduate School of Medical and Dental Sciences
  • Kawabe Jun-ichi
    Department of Cardiovascular Regeneration and Innovation, Asahikawa Medical University
  • Takahashi Fumihiko
    Department of Internal Medicine, Hokkaido Prefectural Haboro Hospital
  • Kobayashi Motoi
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Yamauchi Atsushi
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Sasaki Yukie
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Sakamoto Naka
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Ota Hisanobu
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Tanabe Yasuko
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Takeuchi Toshiharu
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Takenaka Toshihiro
    Division of Cardiac Repair and Regeneration, Graduate School of Medical and Dental Sciences, Kagoshima University
  • Kikuchi Kenjiro
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Hasebe Naoyuki
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Nakagawa Naoki
    Division of Cardiology, Nephrology, Pulmonology and Neurology, Department of Internal Medicine, Asahikawa Medical University
  • Maruyama Hiroki
    Department of Clinical Nephroscience, Niigata University Graduate School of Medical and Dental Sciences
  • Ishihara Takayuki
    Department of Clinical Nephroscience, Niigata University Graduate School of Medical and Dental Sciences

書誌事項

タイトル別名
  • Identification of a Novel <i>α</i>-Galactosidase A Missense Mutation (G195V)
公開日
2011
資源種別
journal article
DOI
  • 10.1536/ihj.52.308
公開者
一般社団法人 インターナショナル・ハート・ジャーナル刊行会

この論文をさがす

説明

Fabry disease is an X-linked lysosomal storage disorder caused by mutations of the α-galactosidase A gene (GLA), and the disease is a relatively prevalent cause of left ventricular hypertrophy mimicking idiopathic hypertrophic cardiomyopathy. We assessed clinically 5 patients of a three-generation family and also searched for GLA mutations in 10 family members. The proband had left ventricular hypertrophy with localized thinning in the basal posterior wall and late gadolinium enhancement (LGE) in the near-circumferential wall in cardiovascular magnetic resonance images and her sister had vasospastic angina pectoris without organic stenosis of the coronary arteries. LGE notably appeared in parallel with decreased α-galactosidase A activity and increased NT-pro BNP in our patients. We detected a new GLA missense mutation (G195V) in exon 4, resulting in a glycine-to-valine substitution. Of the 10 family members, 5 family members each were positive and negative for this mutation. These new data extend our clinical and molecular knowledge of GLA gene mutations and confirm that a novel missense mutation in the GLA gene is important not only for a precise diagnosis of heterozygous status, but also for confirming relatives who are negative for this mutation.

収録刊行物

  • International Heart Journal

    International Heart Journal 52 (5), 308-311, 2011

    一般社団法人 インターナショナル・ハート・ジャーナル刊行会

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