2. Alright tell
me how you
want to
change…!

3. • It is a technique for correcting
defective genes that are responsible
for disease development

4.  1. A normal gene inserted to
compensate for a nonfunctional gene.
 2. An abnormal gene traded for a
normal gene
 3. An abnormal gene repaired through
selective reverse mutation
 4. Change the regulation of gene pairs

5. 
 · 1970s Gene surgery proposal
 • 1980s Great momentum to GT
 • 1983 Gene therapy Lesch-Nyhan disease GT
 • In 1988 (OTA)US Differentiate in somatic &
germ LINE gene therapy
 • September 14, 1990 ADA deficiency GT(W.
French Anderson and colleagues)successful
 • IN 1991 US govt.$58 million for gene therapy
research
 • Oct.1999.Jesse Jelsinger first fatality in human
gene EXP :Multiple organ failure.(Ornithine
Trancarbamylase Deficiency)
 •2002 X-SCID: Fatal Leukemia

6.  • The first gene therapy was performed
on September 14th, 1990
 • Ashanti DeSilva was treated for SCID
 • Sever combined immunodeficiency
 Doctors removed her white blood
cells, inserted the missing gene into the
WBC, and then put them back into her
blood stream
 . • This strengthened her immune system
 • Only worked for a few months

7.  • A vector delivers the therapeutic gene
into a patient’s target cell
 • The target cells become infected with
the viral vector
 • The vector’s genetic material is
inserted into the target cell
 • Functional proteins are created from
the therapeutic gene causing the cell to
return to a normal state

9.  Somatic gene therapy:
 a. Ex vivo gene therapy
 b. In vivo gene
 Germline therapy & embryonic gene
therapy

12.  1. Germline gene therapy refers to the
permanent transfer of a gene into sperm
or egg cells.
 2. Embryonic gene therapy refers to the
permanent transfer of a gene into the
cells of an early embryo, just after the
sperm and egg unite
 .3. In both cases, the delivered gene
would become a permanent part of
cells in the resulting adult.

14.  • Calcium phosphate
 • Gold nanoparticles (1 to 3 micrometer)
:800psi
 • Electroporation
 • Microinjection
 • Chitosan
 • Elevated temprature
 • DEAE dextran
 • Cationic liposome
 • Activated dendrimer

18. • Short Lived
– Hard to rapidly integrate therapeutic DNA into genome and rapidly dividing
nature of cells prevent gene therapy from long time
– Would have to have multiple rounds of therapy
• Immune Response
– new things introduced leads to immune response
– increased response when a repeat offender enters
• Viral Vectors
– patient could have toxic, immune, inflammatory response
– also may cause disease once inside
• Multigene Disorders
– Heart disease, high blood pressure, Alzheimer’s, arthritis and diabetes are
hard to treat because you need to introduce more than one gene
• May induce a tumor if integrated in a tumor suppressor
gene because insertional mutagenesis

21.  • Neurogenic disease may be cured e.g.
Alzeimer & Parkinsons Disease (life
threatening diseases)
 • Difference types of Cancer gene therapy
trials may get success in future
 • Germ cell gene therapy trials may get
success in curing hereditary diseases
 • Pharmacogenetics & Geneticular
molecular biotechnology
 • Applicable in livestock species.
 • GT: cautiously & seriously evaluate