This document discusses gene therapy, including: - Gene therapy involves inserting a normal gene to replace an abnormal gene responsible for disease. - There are two main types: germ line therapy affects future generations, while somatic therapy only affects the individual. - The basic process involves researching the defective gene, copying the normal gene, and inserting it into cells. - Gene therapy could potentially replace defective genes, deliver genes to treat cancer or provide immunity, and stimulate tissue growth or healing. However, it also faces challenges like high costs and ensuring genes only target intended cells. Examples where it has been used include hemophilia, blindness, cancer, and Parkinson's disease.

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