title: Amyloid heart disease: genetics translated into disease-modifying therapy.
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.
authors.affiliation: Department of Cardiovascular Medicine, Cleveland Clinic Foundation, Cleveland, Ohio, USA.
authors.forename: Brett W
authors.initials: BW
authors.lastname: Sperry
authors.affiliation: Cleveland Clinic Foundation, Center for Clinical Genomics, Cleveland, Ohio, USA.|Department of Cardiovascular Medicine, Cleveland Clinic Foundation,, Ohio, USA.
authors.forename: W H Wilson
authors.initials: WHW
authors.lastname: Tang
mesh_terms: D000686:Amyloidosis / Q000235:genetics* / Q000628:therapy; D009202:Cardiomyopathies / Q000235:genetics* / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans
keywords: Amyloid heart disease; gene silencing; restrictive cardiomyopathy