Challenges faced in gene therapy

Gene therapy

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: Somatic cell gene therapy germline therapy

4. Challenges Faced In Gene Therapy We have faced following Challenges during Gene Therapy: Gene delivery and activation Immune response Disrupting important genes in target cells 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.