Gene and stem cell therapy are still in their infancy, and the going is
difficult, but progress is steady. These two forms of therapy involve the
replacement of cell components or whole cells within the body.
One area where gene therapy can be useful is in getting rid of extracellular
and intracellular junk.
Inside the cell is the lysosome, a special vessel containing an enzyme that
breaks down junk inside and outside the cell. Some humans born without this
enzyme suffer from Gaucher’s disease, a deficiency in the enzyme-producing
gene. Doctors use gene therapy to replace the defective genes, triggering the
cell to produce lysosomal enzyme.
De Grey and SENS scientists predict that in the near future, similar
techniques will allow doctors to replace defective genes, boosting their
ability to fix damage from oxidation. Gene therapy can wipe out
artheriosclerosis, brain degeneration, and macular degeneration, among many
illnesses.
Perhaps the most daring application for gene therapy is WILT, or whole-body
interdiction of lengthening of telomeres. If it works, it can be the best cure
for cancer.
The goal of WILT is to prevent the production of the enzyme telomerase,
which maintains telomeres and keeps them long. Telomeres, found at the end of
each chromosome, get shorter every time the cell divides.
Experiments show that telomere length is related to a cell’s mortality. When
a person has cancer, the telomeres behave strangely; they don’t get shorter
when they divide. Cancer cells take a long time to die, so they just multiply
and grow in size, producing a tumor.
Scientists hope that gene therapy can eliminate the gene that makes
telomerase, making cells die before they reach cancerous proportions.
However, a new set of problems arises. One is how to insert the engineered
gene into the body so that a person’s cells will not contain telomerase. Stem
cell therapy is one solution.
Gene therapy allows scientists to engineer a gene not to have telomerase.
They would then grow stem cells with that gene and replace the person’s
defective stem cells, through a bone marrow transplant, with those containing
the reengineered gene.
In addition, stem cell therapy can replace blood stem cells to contain
enzymes designed to slow down and fix damage caused by oxidation.