GENE THERAPY

1. GENE
THERAPY
SUSHAN GAIRE
B.PHARM 2ND
SEMSTER

2. CONTENTS
• INTRODUCTION TO GENE THERAPY
• TYPES OF GENE THERAPY
A. GERM LINE GENE THERAPY
B. SOMATIC GENE THERAPY
• BASIC PROCESS OF GENE THERAPY
• USE OF GENE THERAPY
• DISADVANTAGE OF GENE THERAPY
• EXAMPLES OF GENE THERAPY

3. WHAT IS GENE THERAPY?
 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.

4. Inactivating or “knocking out” a mutated
gene that is functioning improperly.
Introducing a new gene into the body to help
fight a disease.

5. TYPES OF GENE THERAPY
Germ
Line
Gene
Therapy
Somatic
Gene
Therapy

6. GERM LINE GENE THERAPY
Therapeutic genes transferred into the germ
cells.
Heritable and passed on to later generations.
EG:Genes introduced into eggs and sperm.

7. SOMATIC GENE THERAPY
Therapeutic genes transferred into the
somatic cell.
Will not be inherited later generations.
Eg: Introduction of genes into bone marrow
cells,blood cells,skin cells etc.

8. BASIC PROCESS OF GENE
THERAPY
Doing research to find the gene involved in
genetic disorder.
Cutting out the normal allele.Special enzyme
like proteins which catalyze or speed up
chemical reactions inside our body are used
to do this.
Making many copies of the allele.
Putting copies of the normal allele into the
cell of the person who have genetic disorder.

9. USE OF GENE THERAPY
Ability to replace defective genes.
Deliver genes that speed the destruction of
cancer cells & supply genes that causes
cancer cells to revert back to normal cells.
Deliver bacterial or viral genes as a form of
vaccination.
Provide genes that promotes or impede the
growth of new tissue.
Deliver genes that stimulate the healing of
damaged tissue.

10. DISADVANTAGES OF GENE
THERAPY
The genetic testing, research and screening in
finding the availability of certain gene is very
controversy.
The cost is very high and requires latest
technology with skilled knowledge.
Possibility of DNA entering cells other than
target cells with unknown effects.

11. EXAMPLES OF GENE THERAPY
Some examples of GT are:-
Treatment of Hemophilia.
Treatment of Hereditary blindness
Treatment of Cancer
Treatment of Parkinson's disease