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Gene Therapy by Lekhan
This document discusses gene therapy, which involves inserting normal genes into patients to replace abnormal genes that cause diseases. The most common form replaces a mutated gene with a healthy copy. Gene therapy can be used for diseases like severe combined immunodeficiencies, haemophilia, Parkinson's disease, cancer, and cystic fibrosis. There are two main types: germline gene therapy results in permanent changes that are inherited, while somatic gene therapy only affects the patient's cells and is not passed to offspring. The document outlines the process researchers use to design gene therapies.
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Gene Therapy by Lekhan
- 1. GUIDED BY PRESENTEDBY Dr.RASHMI SHRIVASTAVA Assist.Prof. Department of zoology LEKHAN LODHI M.Sc II nd Sem Department Of Zoology GENE THERAPY
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- 3. GENE THERAPY Anexperimental technique for correcting defective genes that are responsible for disease development. The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene.
- 4. OTHER APPROACHES USED……….. Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating or “knocking out” a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease.
- 5. GENE THERAPY FORDISEASES…….. Diseases like........... Severe Combined Immune-Deficiencies (SCID) Haemophilia Parkinson’s Disease Cancer Cystic fibrosis
- 6. TYPES OF GENETHERAPY GERM LINE GENE THERAPY SOMATIC GENE THERAPY
- 7. GERM LINE GENETHERAPY….. Results in permanent changes Potential for offering a permanent thereptic effect for all who inherit the target gene Targeting of germ cells makes the therapy heritable.
- 8. SOMATIC GENE THERAPY Affects only the targeted cells in the patient & is not passed to future generations. Appropriate & acceptable for many disorders including Cystic fibrosis, muscular dystrophy, cancer
- 9. Ex-vivo Cells aremodified outside the body and then transplanted back in again Called ex-vivo because the cells are targeted outside the body In-vivo Genes are changed in cells when the cells are still in the body Called in-vivo because the gene is transferred to cells inside the patient’s body TYPES OF SOMATIC GENE THERAPY
- 12. ADVANTAGES OF GENETHERAPY Give a chance of a normal life to baby born with genetic diseases Give hope of healthy life to cancer patients For certain disease that do not have any cure except gene therapy , it could save many lives
- 13. TO DESIGN GENETHERAPY…….. A researcher must………. Identify the genes responsible for the disorder Make copies of the normal gene Insert the copies into vectors Infect the affected cells with the vectors Activates the gene so that transcription and translation takes place.
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