Overall strategies of Myocardial Gene
Transfer
The overarching concept of cardiac gene therapy is simple: replace or
remove a disease-causing gene at the level of the myocardium, thereby
eliminating a fundamental incipient for a given condition. In practice,
there is an array of selection criteria and obstacles to consider. For
any gene therapy to be successful, the gene(s) of interest must not only
be delivered but also expressed at adequate concentrations in the target
tissue bed. The tools used to accomplish this gene transfer are known as
vectors. The ideal vector manifests tissue selectivity, low
immunogenicity, adequate packaging capacity, and a durable level of gene
expression. To date, current vector options incorporate some, but not
all of these attributes. Vectors can be described in two broad
categories: viral (gene transduction) and non-viral (transfection).
Following selection, the vector of choice may be delivered to the
myocardium through a variety of techniques over a spectrum of
invasiveness and specificity. There is no single optimal combination of
the above factors, rather, it is necessary to understand the appropriate
applications and limitations of each.