Transcript Gene Therapy: “Mr. Fix-it” for Cells

Gene Therapy: “Mr. Fix-it” for
Cells
What is a Gene?
• The “blueprint” for our bodies comes in the
form of chromosomes, made out of DNA
Question: What does DNA stand for?
• DNA encodes lots and lots of different genes
(pronounced “jeans”), which specify hair and
eye colour, height, size of feet, etc.
Genes and Diseases
• “faulty” or missing genes cause disease
• Genetic conditions used to be
considered a “life sentence”
Is this still the case??
Gene Therapy
• Gene Therapy: Treating a genetic
disease by inserting a healthy version of
the missing or defective gene into a
patient’s cells
Could it be True?
True or False:
1. Scientists have used gene therapy to
successfully restore sight to blind dogs
2. Scientists used gene therapy to make mice
“smarter”
What do you think?
How Can You Get Genes into
Cells?
• Viruses (including perhaps HIV)
Viral genes
inserted into
target cell
http://www.tulane.edu/~dmsander/WWW/224/Classification224.html
How Can You Get Genes into
Cells?
• Other weird and wacky ways of putting
healthy genes into cells…..
– Liposomes (“mini cells”)
Liposome
– Electroporation (making cell membrane
more “holey” to allow genetic material to
enter cytoplasm)
Does Gene Therapy Work?
• Success in treatment
of SCID in two infants
– “bubble boy”
syndrome
• Viruses used to
deliver functional
gene
Movie: Bubble Boy (2001)
Another Question……
Which of the following diseases are current or potential targets of
gene therapy?
a. Cancer
b. AIDS
c. Diabetes
d. Parkinson’s
Disease
Ethical Considerations
(William Bates, 2001)
What Lies Ahead?
• Formation of groups with 4 or 5
students/group to participate in:
– Gene therapy scavenger hunt
– Gene therapy debate