GENE THERAPY [Autosaved].pptx A brief explanation about Gene Therapy

A way to treat genetic disorder

1. GENE THERAPY Gene therapy aims to fix a faulty gene or replace it with a healthy gene to try to cure disease or make the body better able to fight disease

2. CONTENTS Gene Therapy History Types Gene delivery Vectors Advantage Disadvantage

3. What is Gene Therapy Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use . Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Gene therapies can work by several mechanisms: Replacing a disease-causing gene with a healthy copy of the gene Inactivating a disease-causing gene that is not functioning properly Introducing a new or modified gene into the body to help treat a disease Gene therapy products are being studied to treat diseases including cancer, genetic diseases, and infectious diseases.

4. Process of gene therapy in the figure, the cell with the defective gene is injected with a normal gene which helps in the normal functioning of the cell. This technique is employed mainly to fight against the diseases in the human body and also to treat genetic disorders. The damaged proteins are replaced in the cell by the insertion of DNA into that cell.  For example,  cancer  cells contain faulty cells which are different from the normal cells and have defective proteins. Hence, if these proteins are not replaced, this disease would prove to be fatal.

5. History of Gene Therapy

6. Types of Gene Therapy Somatic Gene Therapy Germline Gene Therapy

7. Somatic Gene Therapy This type usually occurs in the somatic cells of human body. This is related to a single person and the only person who has the damaged cells will be replaced with healthy cells. In this method, therapeutic genes are transferred into the somatic cells or the stem cells of the human body. This technique is considered as the best and safest method of gene therapy.

8. Germline Gene Therapy It occurs in the germline cells of the human body. Generally, this method is adopted to treat the genetic, disease causing-variations of genes which are passed from the parents to their children. The process involves introducing a healthy DNA into the cells responsible for producing reproductive cells, eggs or sperms . Germline gene therapy is not legal in many places as the risks outweigh the rewards.

9. Difference between Somatic and Germline gene therapy

10. Gene Delivery Gene delivery is the process of introducing foreign genetic material, such as DNA or RNA, into host cells.

11. Vectors: Tools for Gene Delivery What is vector in Gene therapy The system used to deliver genetic material is known as a vector. Think of a vector as a microscopic delivery truck that transports packages (genetic material) to specific locations (target cells). There are two types of vectors, viral and non-viral. Two main classes Viral vector Non viral vector

12. Viral vectors used in Gene Therapy There are several types of viral vectors that can be used to deliver nucleic acids into the genetic makeup of cells including Adeno-Associated viral (AAV) Adenovirus Retrovirus Lentivirus Herpes simplex Alphaviruses Flaviviruses Rhabdoviruses Measles virus

13. Process of Gene delivery using viral vector

14. Non viral vector used in Gene Therapy Physical methods: Electroporation Microinjection Gene gun or biolistic particle delivery system Magnetofection Chemical method DNA transfer by calcium phosphate method Liposome medicated transfer

15. Disease cured by Gene Therapy

16. Advantage of Gene Therapy Provides options. Gene therapy can potentially cure someone of a disease, especially in instances where no other medications have worked.  Only has to be given one time. Existing gene therapies are given as a  one-time dose . This is appealing compared to some other treatments that require multiple doses. Long-lasting effects.  Gene therapy has the potential to get rid of a person’s symptoms for life. Gene therapy can give many people a better quality of life. Positive effects passed down through generations. If you remove a faulty gene from a parent , they won’t transfer this gene to their kids. Rapidly-changing technology. Gene therapy is able to exist because of massive advances in technology over the past few decades. Since technology continues to advance, we can expect gene therapy to continue to improve. We can also expect more gene therapies to gain FDA approval.

17. Disadvantage of Gene Therapy Expensive. Gene therapy can be extremely pricey, making it inaccessible for some people. For example,  Zolgensma , a gene therapy to treat spinal muscular atrophy, is the most expensive medication in the U.S . It costs $2.1 million for a course of treatment. Experimental.  Gene therapy is relatively new and there’s still a lot about it that we don’t know. Right now, there are no guarantees. Exactly how successful gene therapy will be is still uncertain. Potentially dangerous.  There’s some concern that if gene therapy reaches other cells in the body besides the faulty cell, it could have harmful effects. There’s also concern that reproductive cells could be changed without detection, and these could be passed on to people’s children. Ethical issues . Some people have ethical issues with gene therapy. For example, some wonder who will decide what “good” and “bad” uses of gene therapy are? And will gene therapy make us less accepting of people who are different?

18. Thanks for watching