Transcript Experimental Gene Therapy Use On Humans

Experimental Gene Therapy
Use On Humans
What is gene therapy?
Gene therapy is a method of curing
genetic disorders by introducing
functioning genes into a patient’s body.
These functioning genes are supposed to
correct and replace the defective genes
responsible for genetic disorders.
There are two types of gene therapy.
Somatic gene therapy and germ-line gene
therapy.
Somatic gene therapy Vs.
Germ-line gene therapy
Somatic gene therapy treats patients by
introducing the genes to body cells like
bone marrow or blood cells.
Germ-line gene therapy targets egg and
sperm cells. It is medically impossible at
this moment. This type of gene therapy
would pass the inserted gene to future
generations.
How does gene therapy work?
Genes enter the body of a patient through a
carrier molecule called a vector. Vectors are
usually viruses that have been modified so that
they don’t harm the patient.
Common viruses used as vectors: retrovirus,
adenovirus, adeno-associated virus, herpes
simplex virus, and poxvirus.
Vectors are either injected or given through an
IV.
There are nonviral ways of delivering genes.
Quick history lesson, SON!
Concept of gene therapy originated in the 60s
and early 70s.
On September 1990, the first gene therapy
procedure was approved.
In 1999, Jesse Gelsinger, a gene therapy clinical
trial patient, died four days after beginning his
treatment from multiple organ failures.
In 2008, the first gene therapy trial testing for
inherited blindness was shown to improve sight.
Safety
Because of what happened in 1999, the FDA
placed a temporary halt on all retroviral vectors
used in blood stem cells.
FDA oversees and regulates all gene therapy
products and research in the US.
The National Institutes of Health determine
whether or not a clinical trial raises any ethical,
safety, or medical concerns.
The Institutional Review Board and the
Institutional Biosafety Committee must authorize
each gene therapy clinical trial before it is put
into action.
Ethical Issues
Germ-line gene therapy passes genes to
future generations. Is it right to decide
what genes should be passed on?
Is it right to use to germ-line gene therapy
to improve human traits?
Is somatic gene therapy more or less
ethical than germ-line gene therapy?
My viewpoint
Germ-line gene therapy should be hands
off, with the exception of using it to get rid
of genetic disorders that run in families.
Gene therapy should be used to treat
serious diseases that have no other
treatments.