GENE THERAPY

INTRODUCTION OF GENE THERAPY, HISTORY OF GENE THERAPY, Process of gene therapy, Methods of gene therapy, Ex vivo gene therapy , In Vivo Gene Therapy , Uses of gene therapy, Target sites for Gene Therapy , Vectors for gene therapy , Viral Vectors, Non Viral Vectors,

1. Amity University Jharkhand Assignment topic:- GENE THERAPY Subject:- Basic Biotechnology Submitted by :- Name:- Swati Suman Enrolment no.:- A35204418013 Program :- Bsc .Biotechnology ( 1 semester)

2. Gene Therapy

3. Acknowledgement I would like to express my special thank to my subject teacher Mrs. Rajani Sharma for giving me this golden opportunity of making project on the topic GENE THEREPY . And also my family and friends for helping me in completing this beautiful assignment. Thank you every one.

4. What is gene therapy? Gene therapy is a novel treatment method which utilizes genes or short oligonucleotide sequences as therapeutic molecules, instead of conventional drug compounds. This technique is widely used to treat those defective genes which contribute to disease development. In gene therapy, DNA encoding a therapeutic protein is packaged within a "vector", which transports the DNA inside cells within the body. The disease is treated with minimal toxicity, by the expression of the inserted DNA by the cell machinery.

5. Process of gene therapy

6. Methods of gene therapy. There are mainly two approaches for the transfer of genes in gene therapy: 1. Transfer of genes into patient cells outside the body (ex vivo gene therapy) 2. Transfer of genes directly to cells inside the body (in vivo).

7. Gene Therapy using autologous cells: Cells are used, i.e. cells are removed from the patient, cultured in vitro, before being returned to the patient’s body. In this figure in vivo and ex vivo gene therapy is diagrammatically explained.

8. Ex vivo gene therapy

9. In Vivo Gene Therapy

10. Uses of gene therapy Medical Conditions for Which Gene Therapy Is Being Studied:- ADA deficiency Hemophilia AIDS Liver cancer Asthma Lung cancer Brain tumor Melanoma Breast cancer Muscular dystrophy Colon cancer Neurodegenerative conditions Diabetes Ovarian cancer Heart diseases Prostate cancer

11. Target sites for Gene Therapy Therapeutic genes have to be delivered to specific target sites for a specific type of disease. This table describes the list of such disease and their target sites for gene therapy.

12. Vectors for gene therapy Vectors for gene therapy can be classified into two types: 1. Viral vectors 2. Non-viral

13. Viral Vectors Adenoviral vectors Retroviral Vectors Adeno- Associated Virus (AAV) Herpes simplex virus vectors Baculovirus Simian Virus 40 Vectors (SV40)

14. Adenoviral vectors

15. Retroviral Vector

16. Non Viral Vectors Some non viral methods Direct injection/particle bombardment Microinjection Particle bombardment Electroporation Liposomes Mediated Sleeping Beauty Transposition

17. Liposomes Mediated

18. Sleeping beauty Transposition

19. Advantages of Gene Therapy Gene therapy can cure genetic diseases by addition of gene or by removal of gene or by replacing a mutated gene with corrected gene. Gene therapy can be used for cancer treatment to kill the cancerous cells. Gene expression can be controlled. Therapeutic protein is continuously produced inside the body which also reduces the cost of treatment in long term.

20. GENE THERAPY RISKS The first death associated with gene therapy occurred on September 18, 1999, at the University of Pennsylvania. Jesse Gelsinger was participating in a clinical trial, a biomedical experiment for evaluation of safety and efficiency of a therapy for a disease. Gelsinger, who was 18 years old at the time of the treatment, had a deficiency of ornithine transcarboamylase, an important enzyme in the metabolism of ammonia. Patients with this rare metabolic disorder must maintain a low-protein diet and take a series of medicines to avoid ammonia poisoning in the blood stream. The gene therapy Gelsinger took triggered a chain reaction in his immune system, resulting in hepatic and respiratory failure, and consequently, his death four days after being treated.

21. FINAL CONSIDERATIONS Although the idea of gene therapy has been around for only 20 years, the technique has been drawing a great deal of interest and curiosity through the world. The first trials generated great expectations within the scientific community. Although there have been several disappointments, many believe that it is just a matter of time before the technical and scientific details are mastered and the procedures become routine. This research is being advanced worldwide. In fact, Alain Fischer, a medical doctor in Paris, France, reported the complete cure of two children who had a rare immune deficiency condition.

22. Another promising result from stem cell research has been reported in type-B hemophilia patients at the Children’s Hospital in Philadelphia and at Stanford University, where patients treated with gene therapy presented a reduction in the period for blood coagulation. ADA deficiency, a disease caused by a defective gene for the ADA enzyme present on human chromosome 20 has been a focus for gene therapy in many institutions. In one of the cases, several patients treated with the corrective gene were able to reconstitute their immune systems and are living normal lives out of the isolated bubbles that are needed to maintain an environment free from microbes. The patients started to produce a correct ADAenzyme after receiving the gene therapy.

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