Targeted gene transfer in heart failure: implications for novel gene identification

Heart failure remains an intractable disease with epidemic proportions in the Western World. While progress in conventional treatment modalities for congestive heart failure is making steady and incremental gains to reduce this disease burden, there remains a need to explore new potentially therapeutic approaches. Gene therapy, for example, was initially envisioned as a treatment strategy for inherited monogenic disorders. It is now apparent that gene therapy has broader potential that also includes acquired polygenic diseases, such as heart failure. Advances in the understanding of the molecular basis of congestive heart failure, together with the evolution of increasingly efficient gene transfer technology, has placed congestive heart failure within reach of gene-based therapy. In addition, gene-based reconstitution of a normal phenotype allows us to closely examine the behavior of a large number of transcripts as the heart fails and is rescued by genetic manipulations.