3. Gene therapy
Gene therapy is a technique for correcting
defective genes that are responsible for
disease development.
These defective gene causes diseases & illness.
Gene therapy holds promise for treating a wide
range of diseases, such as cancer, cystic
fibrosis, heart disease, diabetes, hemophilia and
HIV.
4. 1. Replacing a mutated gene that causes disease with a healthy copy of the gene.
2. Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
3. Introducing a new gene into the body to help fight a disease.
Gene therapy can be accomplished
by:
6. 1. Somatic gene therapy targets genes in specific
types of cells ( blood cells, bone marrow cells, skin
cells)
2. The edited gene is contained only in the target cell
type . No other types of cells are affected.
3. The edited gene is not passed down to future
generation.
4. This technique is considered as best & safest
method of gene therapy.
Somatic gene therapy
7. 1. Therapeutic genes transferred into the germ
cells.
2. The edited gene is copied in every cell,
including sperm & eggs.
3. The edited gene is passed on to future
generation.
4. This technique is not legal in many places.
Germline gene therapy
8. Ex-Vivo gene therapy
Involves the genetic modification of cells outside of
the body to produce therapeutic factors.
Take out the stem cell from the body
Start the culture the stem cell and produce cell
line of stem cell
Stem cell which have faulty gene introduce a
correct gene of these types by some technique
Then again cell growing
Normal gene replace the faulty gene
Then injected in to the body
Approaches
9. In vivo gene therapy uses viruses or other
methods to deliver genes directly into your
cells.
Normal gene insert in to vector
These vector injected into the body
Vector knows how to go inside the body
cell
Then normal gene replace the faulty gene.
In-vivo gene therapy
10. Gene Therapy for Genetic Disorders
Severe Combines Immune Deficiency (ADS-SCID)
Affected children are born without an effective immune system
and will Succumb to infections.
The disease is caused by a mutation in gene
codes for the enzyme adenosine deaminase (ADA).
Applications of Gene Therapy
11. The therapeutic gene called ADA was introduced into the bone marrow cells of such patients
in the laboratory, followed by transplantation of the genetically corrected cells back to the
same patients.
The immune system was reconstituted.
12. CGD is a genetic disease in the immune system that leads to the patients inability to fight
off bacteria and fungal infections that can be fatal.
Investigators in Germany treated two patients with this disease some of the blood-
making cells are taken from the patient.
The normal gene is placed into the cells using special viruses called retroviruses. The cells
are then able to produce the normal protein.
Chronic Granulomatus Disorder
(CGD)
13. Choroideremia is a rare inherited cause of blindness that affects around 1 in 50,000 people.
There is currently no cure.
It is caused by defects in the CHM gene on the X chromosome.
Without the protein produced by the CHM gene, pigment cells in the retina of the eye
slowly stop working, then die off.
Gene therapy trial for inherited
blindness
14. Cancer
Multiple gene therapy strategies have been developed to treat a wide variety of cancers,
including suicide gene therapy, oncolytic and therapeutic gene vaccines.
Two thirds of all gene therapy trials are for cancer and many of these are entering the
advanced stage, including a Phase lll trial.
Additionally, numerous Phase I and Phase II clinical trials for cancers in the brain, skin,
liver, colon, breast and kidney among others, are being conducted in academic medical
technologies and therapeutics developed on-site.
Gene therapy for acquired disease
15. The same gene therapeutic techniques have been applied to treat other
acquired disorders such as viral infections (e.g. influenza, HIV, hepatitis) , heart disease and
diabetes, among others.
Some of these have entered, or will soon be entering, into early phase
clinical trials.
Other acquired diseases
