Gene therapy is an experimental technique that uses genes to treat or prevent disease which allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

1. Gene Therapy
by Prof. Liwayway Memije-Cruz

2. GENETHERAPY“It is
tremendously
rewarding to see
how our work,
starting with the
test tube, can
positively impact
the society, by
creating the
groundwork for
new medical
treatments. “-
Christine Karim

3. What Are Cells?
• the basic building
blocks of all living
things.
• the smallest
structural and
functional unit of an
organism.

4. What are genes?
• the basic physical and
functional units of heredity.
• made up of DNA. Some genes act as instructions to
make molecules called proteins.
• Genes vary in size from a few hundred DNA bases to
more than 2 million bases.
• provide the information for the production of specific
proteins and enzymes that make muscles, bones, and
blood, which in turn support most of our body’s
functions, such as digestion, making energy, and
growing.

5. What is Gene Therapy?
• involves the
manipulation of
genes to fight or
prevent diseases.
• it introduces a
"good" gene into a
person who has a
disease caused by a
"bad" gene.

6. How does gene therapy work?
What are the two types of gene
therapy?

7. Somatic Gene Therapy
• involves
introducing a
"good" gene into
targeted cells to
treat the patient
• more
conservative,
safer approach
because it affects
only the targeted
cells in the
patient, and is not
passed on to
future
generations.
• used to correct
the disease
causing genetic
sequence

8. Categories of Somatic Gene Therapy
• ex vivo, which means exterior (where cells are modified
outside the body and then transplanted back in again). In
some gene therapy clinical trials, cells from the patient’s
blood or bone marrow are removed and grown in the
laboratory. The cells are exposed to the virus that is carrying
the desired gene. The virus enters the cells and inserts the
desired gene into the cells’ DNA. The cells grow in the
laboratory and are then returned to the patient by injection
into a vein. This type of gene therapy is called ex vivo
because the cells are treated outside the body.
• in vivo, which means interior (where genes are changed
in cells still in the body). This form of gene therapy is called
in vivo, because the gene is transferred to cells inside the
patient’s body.

9. Ex vivo and In vivo

10. Germline Gene Therapy
• which involves modifying the genes in egg or sperm
cells, which will then pass any genetic changes to future
generations.

11. Possible Effects of Gene Therapy
• The viruses and other agents used to deliver the "good" genes
can affect more than the cells for which they're intended. If a
gene is added to DNA, it could be put in the wrong place,
which could potentially cause cancer or other damage.
• Genes also can be "overexpressed," meaning they can drive
the production of so much of a protein that they can be
harmful.
• a virus introduced into one person could be
transmitted to others or into the environment.

12. References:
• https://kidshealth.org/en/parents/gene-
therapy.html?fbclid=IwAR21RCVn4NR1CYtnOB
u2Y9zih9bH2LfGZmdnk_YvzTC9EydrTuVuXn_
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• https://www.fda.gov/consumers/consumer-
updates/what-gene-therapy-how-does-it-work
• https://jamanetwork.com/journals/jamaotolaryng
ology/article-abstract/621891
• https://www.youtube.com/watch?v=GbJasFgJkL
g&feature=emb_logo