2. GENE
THERAP
Y
Gene Therapy
Before just diving into the main topic let me just tell
you basic detail about gene therapy.
Look at the two words 'Gene' and 'Therapy', which
self explains that there's some therapy for our
genes/gene.
Now, Why would we need a Therapy?
When there's some consequences or some issues
with the gene. Generally, these consequences are -
Addition , deletion or mutation in the normal gene.
Gene therapy is basically a technique which involves
replacement of defective genes with healthy ones in
order to treat the genetic disorder.
2
3. APPLICATION
This method is used to treat diseases such as Cancer, ADA deficiency, cystic fibrosis,
etc.
ADA- Adenosine De-Aminase,
the enzyme is crucial for immunity and the disease is caused by gene deletion.
Similarly with other diseases too. 3
4. HOW GENE THERAPY CURES VIR
DISEASES?
As a First Step towards Gene Therapy, Lymphocytes
from blood from blood of patient are grown in culture
outside the body.
A Functional ADA cDNA using retrovirus is then
introduced in these lymphocytes, which is returned
to the patients.
However, these cells are not immortal. as you know
the life cycle of lymphocytes. The patient requires
periodic infusion of such genetically engineered
lymphocytes 4