GENE THERAPY: PRINCIPLES, PROBLEMS AND PROSPECTS

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GENE THERAPY: PRINCIPLES, PROBLEMS AND PROSPECTS

  1. 1. BCH 491: SPECIAL TOPICS IN BIOCHEMISTRY DEPARTMENT OF BIOCHEMISTRY ISIKAKU, ALBERT TOCHUKWU 2009/163918 SEMINAR TOPIC : GENE THERAPY: PRINCIPLES, PROBLEMS AND PROSPECTS SUPERVISOR : DR. B.C NWANGUMA NOVERMBER, 2012
  2. 2. Genes• The basic unit of heredity• Are carried on a chromosome• Encode how to make proteinDNA RNA protein• Proteins carry out most of the functions of life.• When there is a mutation in the gene, Changes in the conformation and functions of theprotein Diseases
  3. 3. Types Of Gene Therapy somatic gene therapy transfer of gene into somatic cell such as organs germline gene therapy gene transfer into germ cells (sperm and ova)
  4. 4. Fig. 2. Diagram showing ex-vivo method of gene transfer http://wikipedia/gene therapy /method-ex vivo/image
  5. 5. Fig. 3. Diagram showing in-vivo method of gene transfer http://wikipedia/gene therapy /method_in-vivo/image
  6. 6. SOME OF THE VIRAL VECTORS FOR GENE THERAPY
  7. 7. BASIC PRINCIPLE OF GENE THERAPYFig. 4. Gene transfer mechanism http://wikipedia/gene therapy /principle/image
  8. 8. Diseases For Applying Gene TherapyDisease Defect Target cellSevere combined Bone marrow cells orimmunodeficiency T-lymphocytesHemophilia Liver, muscleCystic fibrosis Lung CellsCancer Many cell typesNeurological diseases Parkinson’s/Alzheimer’s Nerve CellsInfectious diseases AIDS, hepatitis B White Blood Cells
  9. 9. PROSPECTS OF GENE THERAPY• Overall impact on patient’s life• Avoidance of readministration• Reduction of effect immunogenicity• Long-term safety, including tumour risk• Plans to demonstrate comparability
  10. 10. CONCLUSIONAll inventions start with great challenges.As the techniques is in its infancy restricted toclinical trials. So with the development ofbetter delivery system and certain othertechnical modifications, gene therapy will bereally a boon to human kind in future.

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