augmentation gene therapy

To be effective, augmentation gene therapy requires
methods that ensure the safe, efficient and stable introduction
of genes into human cells. The production of adequate
amounts of gene products in appropriate cells and tissues is
needed with appropriate control of gene expression and
reliable methods of monitoring therapeutic effects. Before
application of gene therapy to humans, in vitro studies are
needed together with proof of efficiency and safety in animal
models. The possibility of insertional mutagenesis and the
dangers of expressing genes in inappropriate tissues need to be
considered. There may also be immunological reactions
mounted against viral vector material or the gene product itself
if this represents a protein that is novel to the individual being
treated.