1. GENE
THERAPY
Toluwani Ajayi

2. What is
Gene
Therapy?
• Simply the experimental uses of genes to treat or
prevent diseases.(think of it as inserting an extra
storage space in your phone)
• Genetic diseases likeWilsons disease, Cystic Fibrosis,
etc. are diseases that affect the patient when there is
mutation (an error) in the replication of DNA.
• Wilsons Disease

3. Vectors for
gene
therapy:
Virus
Cons
a.The immune system stops it when it is introduced into
the body.
b.The other virus effects (patients get sick)
c. they can only carry limited genes
Pros
a.They are effective at targeting and entering cells
b.They can be modified not to replicate and destroy cells

4. Vectors for
gene
therapy:
Plasmids
Cons
a.They are not effective at targeting and entering cells.
Pros
a.They are effective at carrying a lot of genes
b. the immune system does not block them.

5. Vectors for
gene
therapy:
Virosomes
(liposomes covered
in viral surface
proteins)
Cons
...No veggies...
Pros
a.They can carry a lot of genes
b.They do not get blocked by the immune system
c.Their surface proteins can be changed to match their
target cells

6. Ways of
delivering
genes into a
patients
body
• InVivo:
• Direct introduction of the vectors carrying the genes
into the patients body.There are problems associated
with this way. (Lets go back to the pros and cons of
vectors used in gene therapy).
• ExVivo:
• The genes that are to be introduced into the patient
are firstly inserted into cells that have been extracted
from the patient.These cells are grown in a dish and
later introduced into the patient.This method hardly
triggers any immune response because there are no
aliens introduced into the patient

7. Challenges
with Gene
Therapy
• Gene delivery and
activation
• Immune response
• Disrupting important genes
in target cells
• Commercial viability

8. Successes
with Gene
therapy
• Cancer
• Immune deficiencies
• Hereditary blindness
• Parkinson's disease etc.