Human Gene Therapy

and
Ethical Issues
Gene therapy introduces or
alters genetic material to
compensate for a genetic
 Commonly asked questions regarding gene
therapy:
•How can “good” and “bad” uses of gene therapy be
distinguished?
•Who decides which traits are normal and which
constitute a disability or disorder?
•Will the high costs of gene therapy make it
available only to the wealthy?
•Could the widespread use of gene therapy make
society less accepting of people who are different?
•Should people be allowed to use gene therapy to
Research Issues
 Scientific and ethical discussions about gene
therapy began many years ago, but it was not
until 1990 that the first approved human gene
therapy clinical trial was initiated. This clinical
However, the success of the therapy was
tentative, because along with the gene
therapy the patients also continued
 How do researchers determine
which disorders or traits warrant
gene therapy?
 Unfortunately, the distinction between gene
therapy for disease genes and gene therapy to
enhance desired traits, such as height or eye
color, is not clear-cut. No one would argue
 Even though gene therapy for the correction of
potentially socially unacceptable traits, or the
enhancement of desirable ones, may improve the
quality of life for an individual, some ethicists fear
 Some of the problems of gene
therapy include:
•Short-lived nature of gene therapy - Before gene
therapy can become a permanent cure for any
condition, the therapeutic DNA introduced into target
cells must remain functional and the cells containing
the therapeutic DNA must be long-lived and stable.
Problems with integrating therapeutic DNA into the
genome and the rapidly dividing nature of many cells
●
Immune response - Anytime a foreign object is
introduced into human tissues, the immune
system has evolved to attack the invader. The
risk of stimulating the immune system in a way
●
Problems with viral vectors - Viruses, while
the carrier of choice in most gene therapy
studies, present a variety of potential
problems to the patient --toxicity, immune and
●
Multigene disorders - Conditions or disorders that
arise from mutations in a single gene are the best
candidates for gene therapy. Unfortunately, some
of the most commonly occurring disorders, such as
●
Chance of inducing a tumor (insertional
mutagenesis) - If the DNA is integrated in the
wrong place in the genome, for example in a
tumor suppressor gene, it could induce a tumor.
●
Religious concerns - Religious groups and
creationists may consider the alteration of an
individual's genes as tampering or corrupting
God's work.
Ethical Concerns of Genetic
Enhancement
Genetic enhancement raises a host of ethical,
legal and social questions. What is meant by
normal? When is a genetic intervention
"enhancing" or "therapeutic?" How should the
That is, will genetic enhancement be available
to all or only to the few who can afford to
purchase it using their personal finances?
These questions relate to the two major
 Some have speculated that genetic
enhancement might affect human evolution.
Philosophical and religious objections also have
been raised, based on the belief that to
 Geneticists have countered that the power to
control human evolution is unlikely, as the
evolution of the human species is a nonrandom
change in allelic frequencies resulting from
Ethical and social concerns center not so
much on the improvement of traits for
alleviation of deficiencies or on the
reduction of disease risk, but on the
Although the distinctions between cure and
enhancement might be obvious to some,
they can lose meaning in medical practice
or in formulating health policy. For
 Prepared by:

Ms. Christine T. Manansala

Q3A BS- Biology