title: S100A1 gene therapy for heart failure: a novel strategy on the verge of clinical trials.
abstract: Representing the common endpoint of various cardiovascular disorders, heart failure (HF) shows a dramatically growing prevalence. As currently available therapeutic strategies are not capable of terminating the progress of the disease, HF is still associated with a poor clinical prognosis. Among the underlying molecular mechanisms, the loss of cardiomyocyte Ca(2+) cycling integrity plays a key role in the pathophysiological development and progression of the disease. The cardiomyocyte EF-hand Ca(2+) sensor protein S100A1 emerged as a regulator both of sarcoplasmic reticulum (SR), sarcomere and mitochondrial function implicating a significant role in cardiac physiology and dysfunction. In this review, we aim to recapitulate the translation of S100A1-based investigation from first clinical observations over basic research experiments back to a near-clinical setting on the verge of clinical trials today. We also address needs for further developments towards "second-generation" gene therapy and discuss the therapeutic potential of S100A1 gene therapy for HF as a promising novel strategy for future cardiologists. This article is part of a Special Section entitled "Special Section: Cardiovascular Gene Therapy".
authors: Laboratory for Molecular and Translational Cardiology, Center for Molecular and Translational Cardiology, Department of Internal Medicine III, University of Heidelberg, Heidelberg, Germany.
authors: David
authors: D
authors: Rohde
authors: Henriette
authors: H
authors: Brinks
authors: Julia
authors: J
authors: Ritterhoff
authors: Gang
authors: G
authors: Qui
authors: Shumei
authors: S
authors: Ren
authors: Patrick
authors: P
authors: Most
mesh_terms: D000818:Animals; D004734:Energy Metabolism; D015316:Genetic Therapy / Q000379:methods*; D006333:Heart Failure / Q000235:genetics / Q000628:therapy*; D006801:Humans; D009418:S100 Proteins / Q000235:genetics*
chemical_list: D009418:S100 Proteins; C110294:S100A1 protein