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  • Is the most common approach
    The abnormal gene would be swapped by homologous recombination
    Would cause a return to normal function
    Control expression of genes. Similar to epistasis, when one gene affects the expression of another gene.
  • A vector is a carrier molecule, usually a virus
    The target cells are usually in the liver or lung

    1. 1. Gene Therapy PRESENTED BY:SURAYYA MUHD LAMIDO STUDENT NO: 20122555 ENVS 525 1st APRIL,2013
    2. 2. DEFINATIONS OF TERMS  GENETICS: is the science of genes, heredity, and variation in living organisms.  GENES: The basic biological unit of heredity. Gene is a unit of heredity that is transferred from a parent to offspring.  CHROMOSOME: A microscopic thread-like structure found within each cell of the body, consisting of a complex of proteins and DNA. Humans have 46 chromosomes arranged into 23 pairs.  THERAPHY: is a treatment intended to relieve or heal a disorder.
    3. 3.  Deoxyribonucleic acid (DNA) : The genetic material in cells that holds the inherited instructions for growth, development, and cellular functioning  RIBONUCLEIC ACID (RNA): is a nucleic acid very similar to DNA. Its structure is the same, although it is usually found singlestranded instead of double stranded like DNA.
    4. 4. Picture of a Chromosome
    5. 5. What is Gene Therapy  Definition: an experimental 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  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.
    6. 6. •Researchers are studying gene therapy for a number of diseases, such as  Severe combined immune-deficiencies (SCID)  Haemophilia  Parkinson's disease  Cancer  HIV
    7. 7. The Beginning…  In the 1980s, Scientists began to look into gene therapy. They would insert human genes into a bacteria cell.  Then the bacteria cell would transcribe and translate the information into a protein  Then they would introduce the protein into human cells 
    8. 8. GERM LINE GENE THERAPY  Result in permanent changes.  Potential for offering a permanent therapeutic effect for all who inherit the target gene.  Possibility of eliminating some diseases from a particular family.  Also raises controversy:  Some people view this type of therapy as unnatural, and liken it to "playing God”.  Others have concerns about the technical aspects.
    9. 9. How It Works
    10. 10. SOMATIC GENE THERAPY  Affects only the targeted cells in the patient, and is not passed to future generations.  Short-lived because the cells of most tissues ultimately die and are replaced by new cells.  Transporting the gene to the target cells or tissue is also problematic.  Appropriate and acceptable for many disorders, including cystic fibrosis, muscular dystrophy, cancer, and certain infectious diseases.
    11. 11. Types of somatic gene therapy
    12. 12. How It Works
    13. 13. Vectors in Gene Therapy  The two major classes of methods :  recombinant viruses – VIRAL VECTOR  naked DNA or DNA complexes – NONVIRAL VECTOR  VI L VECTO R- Viruse s have e vo lve d a way o f RA e ncapsulating and de live ring the ir g e ne s to human ce lls in a patho g e nic manne r. Scie ntists have trie d to harne ss this ability by manipulating the viral g e no me to re mo ve dise ase -causing g e ne s and inse rt the rape utic o ne s
    14. 14. VIRUS  Many GT clinical trials rely on retroviruses or adenoviruses to deliver the desired gene.  Other viruses used as vectors include adeno- associated viruses, lentiviruses, pox viruses, alphaviruses, and herpes viruses.  Differ in how well they transfer genes to the cells they recognize and are able to infect, and whether they alter the cell’ s DNA permanently or temporarily
    15. 15. Non-viral Options           The several methods for non-viral gene therapy are: Injection of naked DNA. Electroporation Gene gum Sonoporation Magnetofection Oligonucleotides Lipoplexes Dendrimers Inorganic nanoparticle.
    16. 16. TYPES OF VIRUS Many GT clinical trials rely on retroviruses or adenoviruses to deliver the desired gene. Other viruses used as vectors include adeno-associated viruses, lentiviruses, pox viruses, alphaviruses, and herpes viruses. Differ in how well they transfer genes to the cells they recognize and are able to infect, and whether they alter the cell’ s DNA permanently or temporarily
    17. 17. Problems with Gene Therapy  Short Lived Hard to rapidly integrate therapeutic DNA into genome and rapidly dividing nature of cells prevent gene therapy from long time  Would have to have multiple rounds of therapy Immune Response  new things introduced leads to immune response  increased response when a repeat offender enters Viral Vectors  patient could have toxic, immune, inflammatory response  also may cause disease once inside Multigene Disorders  Heart disease, high blood pressure, Alzheimer’s, arthritis and diabetes are hard to treat because you need to introduce more than one gene May induce a tumor if integrated in a tumor suppressor gene because insertional mutagenesis     
    18. 18. Current Status  FDA hasn’t approved any human gene therapy product for sale Reasons:  In 1999, 18-year-old Jesse Gelsinger died from multiple organ failure 4 days after treatment for Ornithine transcarbamylase (OTC)  Death was triggered by severe immune response to adenovirus carrier  January 2003, halt to using retrovirus vectors in blood stem cells because children developed leukemia-like condition after successful treatment for X-linked severe combined immunodeficiency disease
    19. 19. Unsuccessful Gene therapies  Jesse Gelsinger, a gene therapy patient who lacked ornithine transcarbamylase activity, died in 1999.  Within hours after doctors shot the normal OTC gene attached to a therapeutic virus into his liver, Jesse developed a high fever. His immune system began raging out of control, his blood began clotting, ammonia levels climbed, his liver hemorrhaged and a flood of white blood cells shut down his lungs.  One problem with gene therapy is that one does not have control over where the gene will be inserted into the genome. The location of a gene in the genome is of importance for the degree of expression of the gene and for the regulation of the gene (the so-called "position effect"), and thus the gene regulatory aspects are always uncertain after gene therapy
    20. 20. Successful One Year Gene Therapy Trial For Parkinson's Disease  Neurologix a biotech company announced that they have successfully completed its landmark Phase I trial of gene therapy for Parkinson's Disease.  This was a 12 patient study with four patients in each of three dose escalating cohorts. All procedures were performed under local anesthesia and all 12 patients were discharged from the hospital within 48 hours of the procedure, and followed for 12 months. Primary outcomes of the study design, safety and tolerability, were successfully met. There were no adverse events reported relating to the treatment.
    21. 21. Recent Developments  Genes get into brain using liposomes coated in polymer call polyethylene glycol  potential for treating Parkinson’s disease  RNA interference or gene silencing to treat Huntington’s   siRNAs used to degrade RNA of particular sequence abnormal protein wont be produced  Create tiny liposomes that can carry therapeutic DNA through pores of nuclear membrane  Sickle cell successfully treated in mice
    22. 22. REFERENCES  Burdette, Walter J. The Basis for Gene Therapy. Springfield: Charles     C Thomas, 2001. Crayton, Stephanie. “First Clinical Trial Of Gene Therapy For Muscular Dystrophy Now Under Way.” Medical News Today. 1 April 2006. University of North Carolina at Chapel Hill. 11 November 2006 <>. Gene Therapy. Human Genome Project Information. 18 November 2005. U.S. Department of Energy Office of Science, Office of Biological and Environmental Research, Human Genome Program. 12 September 2006 <>. McCormack, Matthew P. “Activation of the T-Cell Oncogene LMO2 after Gene Therapy for X-Linked Severe Combined Immunodeficiency.” The New England Journal of Medicine. 346: 1185-1193, Apr 18, 2002. Peel, David. “Virus Vectors & Gene Therapy: Problems, Promises & Prospects.” Virus Vectors & Gene Therapy. 1998. Department of Microbiology & Immunology, University of Leicester. 11 November 2006 <>.