Status of therapeutic gene transfer to treat cardiovascular disease in dogs and cats

Meg Sleeper, Lawrence T. Bish, Mark Haskins, Katherine P. Ponder, H. Lee Sweeney

Research output: Contribution to journalReview article

3 Scopus citations

Abstract

Gene therapy is a procedure resulting in the transfer of a gene(s) into an individual's cells to treat a disease, which is designed to produce a protein or functional RNA (the gene product). Although most current gene therapy clinical trials focus on cancer and inherited diseases, multiple studies have evaluated the efficacy of gene therapy to abrogate various forms of heart disease. Indeed, human clinical trials are currently underway. One goal of gene transfer may be to express a functional gene when the endogenous gene is inactive. Alternatively, complex diseases such as end stage heart failure are characterized by a number of abnormalities at the cellular level, many of which can be targeted using gene delivery to alter myocardial protein levels. This review will discuss issues related to gene vector systems, gene delivery strategies and two cardiovascular diseases in dogs successfully treated with therapeutic gene delivery.

Original languageEnglish
Pages (from-to)131-140
Number of pages10
JournalJournal of Veterinary Cardiology
Volume13
Issue number2
DOIs
StatePublished - Jun 1 2011

Keywords

  • Cardiomyopathy
  • Gene transfer
  • Heart failure
  • Lysosomal storage disease
  • Mucopolysacharidosis

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