gene therapy (introduction, types with examples, advantages, and disadvantages also include limitation of gene therapy.

1. DR . HARISINGH GOUR CENTRAL
UNIVERSITY SAGAR
MADE BY:- NAGENDRA SAHU (MSC 2ND SEM /Y18265012)
GUIDED BY:- DR. RASHMI SRIVASTAVA (DEPARTMENT OF ZOOLOGY)

2. CONTENTS
1. INTRODUCTION
2. VARIETY OF GENE THERAPY
3. TYPES OF GENE THERAPY
I. SOMATIC GENE THERAPY
II. GERM LINE GENE THERAPY
4. LIMITATION OF GENE THERAPY
5. ADVANTAGE AND DISADVANTAGES OF GENE THERAPY

3. WHAT IS GENE THERAPY?
• GENE THERAPY IS THE APPLICATION OF GENETIC PRINCIPLES IN THE TREATMENT OF
HUMAN DISEASE.
• GENE THERAPY TYPICALLY INVOLVES THE INSERTION OF A FUNCTIONING GENE
INTO CELLS TO CORRECT A CELLULAR DYSFUNCTION OR TO PROVIDE A NEW
CELLULAR FUNCTION (CULVER, 1994).

4. • GT IS USED TO IMPROVE A GENETIC DISORDER SUCH AS :-
I. CYSTIC FIBROSIS,
II. SEVERE COMBINED IMMUNODEFICIENCY SYNDROME,
III. MUSCULAR DYSTROPHY,
IV. HEAMOPHILIA
V. CANCERS (PRESENCE OF DEFECTIVE GENES)

5. VARIETY OF GENE THERAPY
 MONOGENIC GENE THERAPY
• PROVIDES GENES TO ENCODE FOR THE PRODUCTION OF A SPECIFIC PROTEIN
(CYSTIC FIBROSIS, MUSCULAR DYSTROPHY, SICKLE CELL DISEASE, HAEMOPHILIA,)
 SUICIDE GENE THERAPY
• PROVIDE ‘SUICIDE’ GENES TO TARGET CANCER CELLS FOR DESTRUCTION
(CANCER)
 ANTISENSE GENE THERAPY
• PROVIDES A SINGLE STRANDED GENE IN AN ’ANTISENSE’ (BACKWARD) ORIENTATION TO
BLOCK THE PRODUCTION OF HARMFUL PROTEINS
(AIDS/HIV)

6. TYPES OF GENE THERAPY
 SOMATIC GENE THERAPY:-
• IN SOMATIC GENE THERAPY, THE THERAPEUTIC GENES ARE TRANSFERRED INTO
THE SOMATIC CELLS,OF A PATIENT. TRANSFER OF A SECTION OF DNA TO ANY
CELL OF THE BODY THAT DOESN’T PRODUCE SPERM OR EGGS. EFFECTS OF GENE
THERAPY WILL NOT BE PASSED ONTO THE PATIENT’S CHILDREN.
I. EX VIVO SOMATIC GENE THERAPY
II. IN VIVO SOMATIC GENE THERAPY

8.  GERM LINE GENE THERAPY:-
• IN GERM LINE GENE THERAPY, GERM CELLS, (SPERM OR OVUM), ARE MODIFIED BY
THE INTRODUCTION OF FUNCTIONAL GENES, WHICH ARE INTEGRATED INTO
THEIR GENOMES.
• TRANSFER OF A SECTION OF DNA TO CELLS THAT PRODUCE EGGS OR SPERM.
EFFECTS OF GENE THERAPY WILL BE PASSED ONTO THE PATIENT’S CHILDREN
AND SUBSEQUENT GENERATIONS.

10. LIMITATIONS OF GENE THERAPY
 SHORT LIVED:-
• IT IS HARD TO RAPIDLY INTEGRATE THERAPEUTIC DNA INTO GENOME AND RAPIDLY
DIVIDING NATURE OF CELLS PREVENT GENE.
 DURATION OF GENE ACTIVITY:
• NON-INTEGRATING DELIVERY WILL BE TRANSIENT.
• INTEGRATED DELIVERY WILL BE STABLE
 PATIENT SAFETY:-
• IMMUNE HYPER-RESPONSIVENESS
 MULTIGENE DISORDERS:-
• HEART DISEASE, HIGH BLOOD PRESSURE, ALZHEIMER’S, ARTHRITIS AND DIABETES ARE
HARD TO TREAT BECAUSE ONE NEED TO INTRODUCE MORE THAN ONE GENE.

11. ADVANTAGES OF GENE THERAPY
1. GENE SILENCING IS A CONCEPT THAT IN ITSELF IS SELF-EFFICIENT FOR MANAGEMENT OF
MANY DISEASES.
2. GENE THERAPY HAS THE POTENTIAL TO ELIMINATE AND PREVENT HEREDITARY DISEASES,
SUCH AS CYSTIC FIBROSIS, AND IS A POSSIBLE CURE FOR HEART DISEASE, AIDS AND CANCER.
3. GIVES AN ADVANTAGE TO A PERSON BORN WITH GENETIC DISORDER TO LIVE LIFE IN A
NORMAL WAY BY REPLACING NON-FUNCTIONAL GENE WITH A FUNCTIONAL ONE.

12. DISADVANTAGES OF GENE THERAPY
1. IRREGULAR IMMUNE RESPONSES.
2. VIRAL VECTORS MAY INTRODUCE TOXICITY.(IMMUNE AND INFLAMMATORY RE-
SPONSES).
3. MULTI-GENE DISORDERS SUCH AS HEART DISEASE, HIGH BLOOD PRESSURE,
ALZHEIMER’S DISEASE.
4. DIABETES CANNOT BE TREATED THROUGH THIS THERAPY.
5. RELIGIOUS CONCERNS.
5. CHANCES OF INDUCING IATROGENIC (PHYSICIAN INDUCED) TUMOURS IN
HUMAN BEINGS.
6. SHORT-LIVED NATURE OF GENE THERAPY.