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Genes.ppt2003
- 1. GENE THERAPY
- 2. Gene Gene Therapy Approaches for gene therapy History How gene therapy is carried out? How it works? Delivery systems Different types of viruses used in gene therapy vectors
- 3. Non-viral Options Disadvantages of gene therapy Disease targets for Gene Therapy oImmunodeficiency disorder oVascular proliferative disorders oLung Diseases oLiver diseases oSkeletal muscle oHemoglobinopathies oCancer oOthers oRare Disease Treated Using Gene Therapy
- 4. GENES Carried on chromosome Basic unit of hereditary Encode how to make protein DNA>RNA>Protein When gene altered cause dysfunction of a protein
- 6. GENE THERAPY It is technique for correcting defective genes that are responsible for disease development Introduction of normal gene into cells that contain defective gene to reconstitute a missing protein product Is used to correct a definitive phenotype so that sufficient amount of a normal gene product are synthesized
- 7. Approaches for Gene Therapy A normal gene inserted to compensate for a nonfunctional gene An abnormal gene traded for a normal gene An abnormal gene repaired through selective reverse mutation Change the regulation of gene pairs
- 8. HISTORY In 1980’s scientists began to look into gene therapy Insert human gene to bacterial cell Bacterial cell would transcribe and translate the information into protein Then they would introduce the protein into human cells
- 9. HOW GENE THERAPY IS CARRIED OUT? Modification of somatic cell by transferring desired gene sequence into the genome Somatic cell necessary to ensure that inserted genes are not carried over to the next generation
- 10. How it works? A vector delivers the therapeutic gene into a patient’s target cell The target cell become infected with the viral vector The vector’s genetic material is inserted into the target cell Functional protein are created from the therapeutic gene causing the cell to return to a normal state
- 13. Delivery systems In vivo:- o Delivery of gene takes place in the body In vitro:- o Delivery takes place out of the body and then cell are placed back into the body
- 14. In vivo technique use viral vector:- – Virus = carrier of desired gene – Virus is usually “crippled” to disable its ability to cause disease – Viral methods have proved to be the most efficient to date – Many viral vectors can stable integrate the desired gene into the target cell’s genome
- 15. In vitro technique usually use manipulation technique:- – Electroporation – Liposome – Calcium phosphate – Gold bullets (fired within helium pressurized gun) – Retrotransposons (jumping genes – early days) – Human artificial chromosomes
- 16. Different types of viruses used in gene therapy vectors Retroviruses:- A class of viruses that can create double standard DNA copies of their RNA genome Copy of its genome can be integrated into the chromosome of host cell HIV virus is retrovirus
- 18. Adenovirus:- A class of viruses with double standard DNA genome that cause respiratory, intestinal, and eye infection in human Virus that cause common cold is adenovirus
- 19. Adeno- associated virus:- A class of small, single, standard DNA viruses that can insert their genetic material at a specific site on chromosome 19
- 20. Herpes simplex virus:- A class of double standard DNA viruses that infect a particular cell type, neurons
- 21. Non-viral Options Direct introduction of therapeutic DNA into target cells The creation of an artificial lipid sphere with an aqueous core Chemically linking the DNA to a molecule that will bind to special cell receptors (less effective )
- 22. Advantage of gene therapy Provide better treatment options for cancer, AIDS and some hereditary disease
- 23. Disadvantages of gene therapy Short-lived nature of gene therapy Immune response Problems with viral vectors Multigene disorders Ethical and legal problems Religious concerns
- 24. Disease targets for Gene Therapy Immunodeficiency disorder :- For the treatment of congenital immunodeficiency disorder illustrate the use of ex vivo gene transfer into hematopoietic stem cell The three important immunodeficiency syndrome that use gene therapy are:-
- 25. Adenosine deaminase deficiency:- o First genetic diseases clinically treated with gene therapy o In children with this disorder leads to the accumulation of deoxyadenosine triphosphate that is toxic to lymphocyte o It develop infections due to the defective cell mediated and humoral immune responses
- 26. Standard therapy is bone marrow transplantation with periodic infusion of PEG-ADA enzyme
- 27. X- linked severe combined immunodeficiency:- Gene therapy for treating children with X-SCID (sever combined immunodeficiency) or the "bubble boy“ Child is born with a very weal immune system Through this therapy, ADA is inserted and then the genetically corrected cells are transplanted back into the child
- 28. Vascular proliferative disorders Atherosclerosis, restenosis after angioplasty, and bypass-graft failure, are leading causes of morbidity and mortality Leads to up-regulated expression of growth factors, cytokines, and adhesion molecules by the vascular smooth-muscle cells
- 30. Lung Diseases Several gene delivery vectors have been applied to treating inherited lung diseases Cystic fibrosis:- Adeno-associated viral vector for delivering CFTR are now in clinical trials
- 31. Alpha-1 Antitrypsin deficiency:- It cause individual to pulmonary emphysema and hepatic chirriosis Recombinant alpha-1 antitrypsin protein is commercially available for human use
- 32. Liver diseases The liver can be afflicted with variety of metabolic, infectious, and neoplastic diseases for which specific molecular intervention can be envisioned
- 33. Skeletal muscle A variety of inherited disorder of muscle including Duchenne muscular dystrophy and the limb girdle muscular dystrophies are prime targets for the development of gene based therapies Adeno- associated virus has been demonstrated to be capable of efficient and stable transduction of adult skeletal muscle
- 34. Hemoglobinopathies Sickle cell disease and the thalassemias are common single gene disorders associated with substantial morbidity and mortality These disorder should be amenable to ex vivo gene transfer into hematopoietic stem cells which then would be used to reconstitute a patient’s bone marrow
- 35. Cancer Multiple strategies have been experimented with to treat different kinds of cancer. These strategies include:- Oncolytic virotherapy Suicide gene therapy Therapeutic gene vaccines Anti-angiogenesis
- 36. Nanotechnology + gene therapy yields treatment to torpedo cancer. A combination of two tumor suppressing genes delivered in lipid-based nanoparticles drastically reduces the number and size of human lung cancer tumors
- 37. Others Chronic Granulomatus Disorder (CGD),in which the process of ADA- SCID is followed to help the patients In Hemophilia, the therapeutic gene is introduced in the patient’s body to cure it.
- 38. Gene therapy is effectively used to treat two adult patients for a disease affecting no lymphocytic white blood cells called myeloid cells Gene Therapy cures deafness in guinea pigs. Thus gene therapy cure deafness
- 39. Neurodegenerative Diseases:- Treatment of neurodegenerative diseases such as Parkinson’s disease and Huntington’s disease
- 40. Rare Disease Treated Using Gene Therapy
- 41. Brain scans of two boys with X- linked adrenoleukodystrophy (ALD):- The scans on the left (A) are from a patient who received gene therapy the one on the right (B) did not The white areas represent the brain damage caused by the disease
- 42. The arrow on the left points to an area that showed some damage, but receded over the course of the therapy When gene therapy started, there were a lot of predictions that this was going to revolutionize medicine. It's been a long time to actually get it to work in humans"
- 43. If the treatment for ALD holds up, it might be the start of that revolution