Gene therapy involves introducing normal genes into cells to replace missing or defective genes and correct genetic disorders. There are two main types of gene therapy: somatic cell therapy and germline therapy. The document discusses four main challenges of gene therapy: 1) delivering genes to the right tissues and cells and ensuring gene activation, 2) immune responses against foreign genes, 3) disrupting important genes in target cells, and 4) the commercial viability of treating rare genetic disorders given the high costs of clinical trials.

1. Presented By:
Syed Dawood Shah
Challenges
Faced In Gene
Therapy
Mirpur University Of Science And
Technology Azaad Kashmir Pakistan

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.