Gene therapy seeks to modify or manipulate genes to treat diseases. There are three main strategies for gene therapy: gene augmentation therapy which introduces a normal gene to compensate for an abnormal gene, targeted killing of specific cells, and targeted inhibition of gene expression. Gene therapy can be somatic, affecting most body cells, or germline, affecting eggs or sperm. Viral vectors are commonly used to deliver genes into cells, but non-viral methods also exist. Gene editing tools like ZFNs, TALENs, and CRISPR-Cas systems can also be used to precisely alter genes.

1. Gen therapy

2. Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the
biological properties of living cells for therapeutic use
Gene therapy is a technique that modifies a person’s genes to treat or cure disease
what is gen therapy?

3. Gene Therapy Strategies
• Gene AugmentationTherapy (GAT)
• Targeted Killing of Specific Cells
• Targeted Inhibition of Gene Expression

4. Gene Augmentation Therapy
(GAT)

5. Targeted Killing of Specific Cells

6. Targeted Inhibition of Gene Expression

7. Type of gen therapy
Types of gene therapy :
1. Somatic gene therapy : most cells of the body
2. Germ line gene therapy : eggs or sperm
Methode gen therapy :
- Ex vivo
- In vivo

8. Methods of gene therapy

9. Methods of gene therapy

10. vectors
In general, a gene cannot be directly inserted into a person’s cell. It must be delivered to the cell using
a carrier, or vector.Vector systems can be divided into:
• ViralVectors
• Non-viralVectors
Currently, the most common type of vectors are viruses that have been genetically altered to carry
normal human DNA

11. Viral
Vectors

12. Viral Vectors

13. Non viral vector

14. Non viral vector

15. Chemical methods nonviral delivery systems

16. Gen editing
tools
There are three families of engineered nucleases
being used:
• Zinc finger nucleases (ZFNs)
• Transcription Activator-Like Effector-based
Nucleases (TALENs)
• CRISPR-Cas system

17. Zinc finger nucleases
(ZFNs)

18. Transcription Activator-Like Effector-based Nucleases
(TALENs)

19. CRISPR-Cas system
Clustered Regularly Interspaced Short Palindromic Repeats CRISPR-Associated Proteins 9