genetic methods used in treatment of diseases

1. Gene therapy
Prepared by:
Samira Fattah Hamid
MSc in medical microbiology-college of medicine-HMU

2. outlines
1. What’s gene ?
2. genetic disorders.
3. Gene therapy.
4. Gene delivery approaches.
5. Concept of Gene Therapy.
6. diseases treated with gene therapy.
7. problems of gene therapy.

3. What’s gene ?
• basic unit of heredity.
• carried on a chromosome.
• are segment of deoxyribonucleic acid (DNA) .

4. What’s gene ?
• A gene consists of a long combination of four
different nucleotide bases:
A (adenine)
C (cytosine)
G (guanine)
T (thymine)

5. • Each person has thousands of genes and
billions of base pairs of DNA which
determine individual characteristics. and
pass genetic traits to offspring.

6. • When genes are altered so that the encoded
proteins are unable to carry out their normal
functions, genetic disorders can result.
• Genetic disorders rarely have effective
treatments, though gene therapy is being tested
as a possible treatment for some genetic
diseases.
genetic disorders

7. Gene therapy
Is the insertion, alteration, or removal of genes
within an individual's cells to treat diseases and
hereditary diseases in which a defective mutant
gene is replaced with a functional one.

8. • Replacing a mutated gene that causes disease with a
healthy copy of the gene.
• Inactivating mutated gene that is functioning improperly.
Approaches for correcting faulty genes:

9. Gene delivery approaches
1-Physical methods
2-Viral vectors

10. Physical methods
 Injection of naked DNA by:
 electroporation.
 Gene gun

11. Viral vectors
 Retroviruses
 Adenoviruses
 Herpes simplex viruses

12. Concept of Gene Therapy
Gene therapy utilizes two theoritically
possible approaches :
2. Somatic gene therapy
1. Germline gene therapy

13. Germline gene therapy
• Involve the genetic modification of germ cells
of an individual and would be carried on to future
generations.
• This would offer the possibility of removing an
inherited disorder from a family line forever.

14. Somatic Gene Therapy
Transfer of a gene into body cells other than
germ cells with effect only on the patient. The
new genetic material cannot be passed on to
offspring

15. Somatic gene therapy can be broadly split
into two categories :
1. Ex vivo
2. In vivo

18. What diseases could be treated with
gene therapy
• About 4000 diseases have been traced to gene
disorders.
• Current and possible candidates for gene therapy include:
cancer
AIDS
Parkinson's disease
Alzheimer’s disease
cardiovascular diseases
and
arthritis

19. • Short-lived nature of gene therapy
• Immune response
• Problems with viral vectors
• Multigene disorders
Problems of gene therapy

20. Summary
• Gene is the basic unit of heredity.
• genetic disorders needs treatment by gene
therapy.
• The aim is by replacing or inactivating a mutated
gene.
• Gene therapy try to treate different types of
diseases.
• During application gene therapy faced some
problems.