title: Genome Editing for the Study of Cardiovascular Diseases.
abstract: The opportunities afforded through the recent advent of genome-editing technologies have allowed investigators to more easily study a number of diseases. The advantages and limitations of the most prominent genome-editing technologies are described in this review, along with potential applications specifically focused on cardiovascular diseases.
  
  
  
  The recent genome-editing tools using programmable nucleases, such as zinc-finger nucleases, transcription activator-like effector nucleases, and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9), have rapidly been adapted to manipulate genes in a variety of cellular and animal models. A number of recent cardiovascular disease-related publications report cases in which specific mutations are introduced into disease models for functional characterization and for testing of therapeutic strategies. Recent advances in genome-editing technologies offer new approaches to understand and treat diseases. Here, we discuss genome editing strategies to easily characterize naturally occurring mutations and offer strategies with potential clinical relevance.
authors.affiliation: Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Alexandra C
authors.initials: AC
authors.lastname: Chadwick
authors.affiliation: Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. kiranmusunuru@gmail.com.|Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA. kiranmusunuru@gmail.com.
authors.forename: Kiran
authors.initials: K
authors.lastname: Musunuru
mesh_terms: D002318:Cardiovascular Diseases / Q000235:genetics*; D064112:Clustered Regularly Interspaced Short Palindromic Repeats; D000072669:Gene Editing / Q000379:methods*; D006801:Humans; D009154:Mutation
keywords: CRISPR/Cas9; Cardiovascular disease; Genome editing; TALEN; ZFN; iPSC