Gene Therapy
Gene therapy
is the introduction of genetic material into cells for therapeutic purposes.
Recent scientific
breakthroughs in the genomics field and our understanding of the important role
of genes in disease has made gene therapy one of the most rapidly advancing
fields of biotechnology with great promise for treating inherited and acquired
diseases.
Many human diseases are
caused by the absence or inappropriate presence of a protein. The protein could then be administered
to patients in order to compensate for its absence. Today, gene therapy is the ultimate method
of protein delivery, in which the delivered gene enters the body's cells and
turns them into small "factories" that produce a therapeutic protein
for a specific disease over a prolonged period.
As gene therapy has moved
from the laboratory into the clinic, several issues have emerged as central
to the development of this technology: gene identification, gene expression
and gene delivery. A number of disease-related genes with direct clinical have
already been identified, and this number is growing as the field rapidly advances.
Genes with broader clinical application are also being utilized to make cells express
immune activating agents locally at the disease site or to become susceptible
to further drug treatment or to immune response recognition.