No Couch Needed: Gene Therapy

The completion of the Human Genome Project in 2004, along with the
sequencing of nonhuman genomes, has spawned an incredible revolution
in the understanding of genetics. Simultaneously, geneticists have raced to
develop medicines to treat and cure diseases caused by genes gone awry.
Gene therapy, treatment that gets at the direct cause of genetic disorders,
is sometimes touted as the magic bullet, the cure-all for inherited diseases
(see Chapter 13 for a partial list) and cancer (see Chapter 14). Gene therapy
may even provide a way to block the genes of pathogens such as the virus
that causes AIDS, providing reliable treatments for illnesses that currently
have none.
Unfortunately, the shining promise of gene therapy has been hampered by a
host of factors including finding the right way to supply the medicine to
patients without causing new or worse problems than the ones being treated.
In this chapter, you examine the progress and perils of gene therapy.