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Amyloid heart disease: genetics translated into disease-modifying therapy
  1. Brett W Sperry1,
  2. W. H. Wilson Tang2,3
  1. 1 Department of Cardiovascular Medicine, Cleveland Clinic Foundation, Cleveland, Ohio, USA
  2. 2 Cleveland Clinic Foundation, Center for Clinical Genomics, Cleveland, Ohio, USA
  3. 3 Department of Cardiovascular Medicine, Cleveland Clinic Foundation,, Ohio, USA
  1. Correspondence to Dr W. H. Wilson Tang, Department of Cardiovascular Medicine, Heart and Vascular Institute9500 Euclid Avenue, Desk J3-4, Cleveland, Ohio 44195; tangw{at}ccf.org

Abstract

Given increased awareness and improved non-invasive diagnostic tools, cardiac amyloidosis has become an increasingly recognised aetiology of increased ventricular wall thickness and heart failure with preserved ejection fraction. Once considered a rare disease with no treatment options, translational research has harnessed novel pathways and led the way to promising treatment options. Gene variants that contribute to amyloid heart disease provide unique opportunities to explore potential disease-modifying therapeutic strategies. Amyloidosis has become the model disease through which gene therapy using small interfering RNAs and antisense oligonucleotides has evolved.

  • Amyloid heart disease
  • gene silencing
  • restrictive cardiomyopathy

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Footnotes

  • Contributors BWS and WHWT drafted and edited the manuscript together.

  • Funding WHWT is supported by grants from the National institutes of Health (R01HL103931) and Collins Family Fund.

  • Provenance and peer review Commissioned; externally peer reviewed.

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