Gene therapy

Gene Therapy (Ex-vivo and In-vivo)

1. GENE THERAPY (ex-vivo & in-vivo) PRESENTED BY, Sharadha M, I M Pharm, Dept.Of Pharmaceutics, JSSCP, Mysore.

2. Contents: 1.Introduction 2.Approaches In Gene Therapy 3.Ex-vivo Gene Therapy 4.In-vivo Gene Therapy 5.Conclusion 6.References

3. Introduction: Gene therapy is the process of inserting genes into existing cells to prevent or cure a wide range of diseases . It is a technique for correcting defective genes responsible for disease development. Types of Gene Therapy: Germ line Gene Therapy Somatic Gene Therapy

4. Approaches In Gene Therapy: Two Types Germ line Somatic Embryo Ex-vivo In-vivo Anti sense

5. Germ Line Gene Therapy Somatic Cell Gene Therapy Therapeutic genes transferred into the germ cells. Therapeutic genes transferred into the somatic cells. E.g. Genes introduced into eggs and sperms E.g. Introduction of genes into bone marrow cells, blood cells, skin cells etc. I t is heritable and passed on to later generations. Will not be inherited later generations. For safety , ethical and technical reasons, it is not being attempted at present. At present all researches directed to correct genetic defects in somatic cells.

6. Ex Vivo Gene Therapy Transplantation or Tissue Grafting Transfer of genes to cultured cells (outside the body) which are then reintroduced into the patient. This technique is used for treating genetic diseases of blood system.

7. Steps: Isolate cells with genetic defect from a patient Grow the cells in culture Introduce the therapeutic genes Select genetically corrected cells and grow Transplant the modified cells to the patient .

8. fig: The procedure for ex-vivo gene therapy

9. Example Of Ex-vivo Gene Therapy Therapy for Adenosine Deaminase Deficiency Therapy for Familial Hypercholesterolemia Therapy for Lesch-Nyhan Syndrome Therapy for Hemophilia

10. In Vivo Gene Therapy The direct delivery of the therapeutic gene (DNA) into the target cells of a particular tissue of a patient constitutes in vivo gene therapy. Gene delivery can be carried out by viral or non- viral vector systems.  This technique is used for treating tissue based genetic diseases

11. fig: In-vivo gene therapy

12. Example Of In-vivo Gene Therapy Therapy for cystic fibrosis In patients with cystic fibrosis, a protein called cystic fibrosis trans-membrane regulator (CFTR) is absent due to a gene defect. In the absence of CFTR chloride ions concentrate within the cells and it draws water from surrounding. This leads to the accumulation of sticky mucous in respiratory tract and lungs . Treated by in vivo replacement of defective gene by adenovirus vector .

13. Conclusion: Theoretically, gene therapy is the permanent solution for genetic diseases. But it has several complexities. At its current stage, it is not accessible to most people due to its huge cost. Gene therapy have the potential to revolutionize the practice of medicine.

14. References: Satyanarayana U, Biotechnology, 1st edition, Book and allied (P) Ltd, Kolkata. Dubey R.C, A textbook of biotechnology, 1st edition(2004), S Chand and company, New Delhi Gupta P.K, Elements of Biotechnology, 1st edition(2001), Rastogi Publications, Meerut.  http :// www.medindia.net/articles/genetherapy_treatment.htm   http://en.wikipedia.org/wiki/Gene_therapy

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