Gene Therapy to Treat Disease - Biotechnology and Your Health: Pharmaceutical Applications - page 95

Stop and Consider
Do you think the current slow pace of gene therapy is a good
thing, or will the caution slow the availability of treatments for
devastating disease?
CONNECTIONS
The potential for gene delivery to actually become gene therapy
seemed very clear decades ago when biotechnology was a young
science, at least in the modern sense. But reality has clouded the
promise. The challenges of delivering the correct gene to the appro-
priate cells and tissue, leading to production of enough protein for
a long period of time, and doing so without causing more harm
than good, have proven to be significant. Laboratory studies, animal
tests, and human trials continue, though the latter are moving more
slowly than in the past because of unforeseen problems. The next
few years will show whether these approaches will prove to be safe
and effective. The increased review, oversight, and caution that were
triggered by Jesse Gelsinger's death and by the leukemia in the
French children may be necessary in order for regulators and the
public to regain confidence in procedures used to protect subjects.
FOR MORE INFORMATION
For more information about the concepts discussed in this chapter,
search the Web using the keywords:
gene therapy , cystic fibrosis , severe combined immunodeficiency disorder
(SCID)