This document summarizes a seminar presentation on gene therapy. It discusses the basic principles of genes and gene therapy, including how genes encode proteins and how mutations can cause disease. It describes the two main types of gene therapy - somatic and germline. It also outlines some viral vectors used, methods of gene transfer like ex vivo and in vivo, diseases that may be treatable with gene therapy, prospects of the approach, and concludes that further development is needed but gene therapy has potential to benefit humanity.

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. Genes
• The basic unit of heredity
• Are carried on a chromosome
• Encode how to make protein
DNA 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 the
protein Diseases

4. 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)

5. Fig. 2. Diagram showing ex-vivo method of gene transfer
http://wikipedia/gene therapy /method-ex vivo/image

6. Fig. 3. Diagram showing in-vivo method of gene transfer
http://wikipedia/gene therapy /method_in-vivo/image

8. SOME OF THE VIRAL VECTORS FOR
GENE THERAPY

9. BASIC PRINCIPLE OF GENE THERAPY
Fig. 4. Gene transfer mechanism
http://wikipedia/gene therapy /principle/image

10. Diseases For Applying Gene Therapy
Disease Defect Target cell
Severe combined Bone marrow cells or
immunodeficiency T-lymphocytes
Hemophilia Liver, muscle
Cystic fibrosis Lung Cells
Cancer Many cell types
Neurological diseases Parkinson’s/Alzheimer’s Nerve Cells
Infectious diseases AIDS, hepatitis B White Blood Cells

12. 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

13. CONCLUSION
All inventions start with great challenges.
As the techniques is in its infancy restricted to
clinical trials. So with the development of
better delivery system and certain other
technical modifications, gene therapy will be
really a boon to human kind in future.