2. What is Gene Therapy?
The introduction of
normal genes into
cells in place of
missing or defective
ones in order to
correct genetic
disorders
3. Types Of Gene Therapy
• There are two types
of gene therapy:
1. Somatic cell gene
therapy
2. germline therapy
4. Challenges Faced In Gene
Therapy
• We have faced following Challenges
during Gene Therapy:
1.Gene delivery and activation
2.Immune response
3.Disrupting important genes in
target cells
4.Commercial viability
5. Gene delivery and activation
• During gene therapy gene must reach
to its destination & it must be
activated.
• Delivering a gene to the wrong tissue
would be inefficient, and it could
cause health problems for the patient.
6. Immune response
• Our immune systems is very good at
fighting to Foreign molecules.
• Immune response is the very important
challenge during gene therapy.
• This situation is overcome by using
immunosuppressive drugs to
temporarily suppress the immune
system.
7. Disrupting important genes in target cells
• Ideal Gene therapy is that an
introduced gene will continue working
for the rest of the patient's life.
• When we introduce foreign gene it
alters the target cell genome and
introduce new challenges.
8. Commercial viability
• Some diseases that can be treated
with gene therapy, such as cancer, are
much more common.
• Many genetic disorders that can
potentially be treated with gene
therapy are extremely rare, some
affecting just one person out of a
million.
• Some gene therapy including taking in
clinical trials necessary for government
approval which are very expensive.