HOW IT WORKS?

• A vector delivers the therapeutic gene into patients target

cell.
• The target cell becomes infected with the viral vector.
• The vectors genetic material is inserted into the target cell
• Functional protein are created from the therapeutic gene
causing the cell to return to the normal state.
 2 TYPES OF PROCESSES
• EX VIVO (outside the body)
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 (inside the body)
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.
DIFFERENCE
 FIRST APPROVED GENE THERAPY
• On September 14, 1990 at the U.S.National Institutes of Health, W.
French Anderson M.D. and his colleagues R.Michael Blaese, M.D., C.
Bouzaid, M.D.,and Kenneth Culver, M.D., performed the first
approved gene therapy procedure on four-year old Ashanthi DeSilva.
Born witha rare genetic disease called severe combined
immunodeficiency (SCID),
• In Ashanthi's genetherapy procedure, doctors removed white blood
cells from the child's body, let the cells grow in the laboratory,
inserted the missing gene into the cells, and then infused the
genetically modified blood cells back into the patient's bloodstream.
 A SUCCESS STORY
• As of early 2007, she was still in good health, and she was attending
college .Some would state that the study is of great importance
despite its indefinite results, if only because it demonstrated that
gene therapy could be practically attempted without adverse
consequences.
 VIRAL VECTORS
• Viral vectors are a tool commonly used by molecular biologists to deliver
genetic material into cells. This process can be performed inside a living
organism.
• Viruses have evolved specialized molecular mechanisms to efficiently
transport their genomes inside the cells they infect.
• Viruses are used as vectors to introduce genetic material inside the bodies
.These viruses are inactivated ,they are not able to reproduce.

SOME VIRAL VECTORS

Adenoviruses
Herpes viruses DNA tumor viruses
Retroviruses RNA tumor viruses
 MAKING SAFE PROTOCOLS

• Low toxicity : The viral vector should have a minimal effect on the
physiology of the cell it infects.
• Stability :Some viruses are genetically unstable and can rapidly
rearrange their genomes. This is detrimental to predictability and
reproducibility of the work conducted using a viral vector and is
avoided in their design
 Gene Therapy for Genetic Disorders
• Severe Combined Immune Deficiency (ADA-SCID)
ADA-SCID is also known as the bubble boy disease. Affected children
are born without an effective immune system and will succumb to
infections outside of the bubble without bone marrow transplantation
from matched donors.
• Chronic Granulomatus Disorder (CGD)
CGD is a genetic disease in the immune system that leads to the
patients' inability to fight off bacterial and fungal infections that can be
fatal.
• Hemophilia
Patients born with Hemophilia are not able to induce blood clots and
suffer from external and internal bleeding that can be life threatening.
 Gene Therapy for Acquired Diseases
• Cancer
Multiple gene therapy strategies have been developed to treat a wide variety
of cancers, including suicide gene therapy, oncolytic virotherapy, anti-
angiogenesis and therapeutic gene vaccines.
Neurodegenerative Diseases
Recent progress in gene therapy has allowed for novel treatments of
neurodegenerative diseases such as Parkinson's Disease and Huntington's
Disease, for which exciting treatment results have been obtained in
appropriate animal models of the corresponding human diseases.
• Other acquired diseases
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.
GENE THERAPY USES AIDS VIRUSES TO FIGHT AIDS
• In the study, immune cells were removed from the patients' bodies
,modified with a disabled AIDS virus known as a lentivirus, and then
intravenously returned .The genetically altered cells disseminated
anti-HIV material and prevented HIV from reproducing( 07November,
2006)