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Gene 
Therapy 
By Pankaj 
Kukreti
CONTENTS 
•DEFINITION 
• TYPES 
• APPROACH 
•METHODS 
• IMPORTANCE 
•DISEASES 
• ETHICS 
• CONCLUSION 
• REFERENCES
WHAT IS GENE THERAPY ? 
• Gene therapy is the insertion of genes into an 
individual’s cells and tissues to treat A 
disea...
TYPES OF GENE THERAPY 
Germ line gene therapy 
• In this case, germ cells, i.E., Sperm or eggs are modified by the 
introd...
NEED OF GENE THERAPY 
• To overcome the disease. 
• Targeting the cells of diseased tissues. 
• Genes can be inserted into...
STRATEGIES OF GENE THERAPY 
•Gene augmentation therapy (GAT). 
• Targeted killing of specific cells. 
• Targeted mutation ...
GENE AUGMENTATION THERAPY 
• For diseases caused by loss of function of a 
gene, introducing extra copies of the normal 
g...
DISEASE CELLS 
GENE X 
NORMAL PHENOTYPE (increase in gene X product)
TARGETED KILLING OF SPECIFIC CELLS 
• Popular in cancer gene therapies. 
• Genes are directed to the target cells and then...
DIRECT KILLING OF DISEASE CELLS 
DISEASE CELLS 
DISEASE CELLS 
CELLS KILLED BY EXPRESSED TOXIN 
CELLS KILLED BY DRUG 
PROD...
TARGETED MUTATION CORRECTION 
• It can be done at different level: 
• At gene level, based on homologous 
recombination. 
...
TARGETED GENE MUTATION CORRECTION 
DISEASE CELLS (mutant gene X) 
NORMAL PHENOTYPE (genetic 
mutation corrected to restore...
TARGETED INHIBITION OF GENE 
EXPRESSION 
• If diseases cells display an inappropriate 
expression of a gene (in case of ma...
Disease cells containing 
mutant or harmful gene 
Block of expression 
of pathogenic gene 
ANTISENSE GENE 
m 
m 
Inhibitio...
METHODS OF GENE TRANSFER 
• VIRAL METHODS 
• RETROVIRUS VECTORS 
• ADENOVIRUS VECTOR 
• HERPES SIMPLEX VECTORS 
• NON VIRA...
Retroviruses are used for Gene Therapy 
LTR gag pol env LTR 
GENE X 
Packaging Signal 
LTR LTR 
10 KB in size 
Gag Pol 
En...
Gene Transfer
BY RETROVIRAL VECTOR
VIRAL METHOD BY ADENOVIRUS 
VECTOR
BY LIPOSOMAL METHOD
GENE THERAPY FOR INHERITED DISORDERS 
• Recessively inherited disorders are 
conceptually the easiest inherited disorders ...
GENE THERAPY SUCCESSES AND FAILURES 
SCID is often called "bubble boy 
disease". SCID became widely known 
during the 1970...
GENE THERAPY TRIALS FOR INHERITED 
DISORDERS 
• DISORDER CELLS ALTERED GENE THERAPY STRATEGY 
ADA DEFICIENCY T-CELLS & HE...
PROBLEMS AND ETHICS 
• SHORT-LIVED NATURE OF GENE 
THERAPY. 
• IMMUNE RESPONSE. 
• PROBLEMS WITH VIRAL VECTORS. 
•MULTIGEN...
CONCLUSIONS 
• It is believed that gene therapy will revolutionize the 
practice of medicine. This technique has the abili...
REFRENCES 
• ASCADI G, DICKSON G, LOVE DR ET AL. NATURE, 352,815-818 
• ANDERSON WF.(1985) J. MED. PHILOSOPH., 10,275-191....
• CULVER KW, BLAESE RM. (1994) TRENDS GENT., 10,174-178. 
CURIEL DT. (1994)ANN.NY ACAD.SCI,716,36-58. 
• ENGLAND SB,NICHOL...
SUSCRIBE US 
FOR MORE 
PRESENTATIONS….
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These slide include gene therapy defines with their types like Germ line gene therapy,Somatic gene therapy.
with Need of Gene therapy
strategies of gene therapy
Methods of Gene transfer & with
GENE THERAPY FOR INHERITED DISORDERS

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Gene therapy

  1. 1. Gene Therapy By Pankaj Kukreti
  2. 2. CONTENTS •DEFINITION • TYPES • APPROACH •METHODS • IMPORTANCE •DISEASES • ETHICS • CONCLUSION • REFERENCES
  3. 3. WHAT IS GENE THERAPY ? • Gene therapy is the insertion of genes into an individual’s cells and tissues to treat A disease, such as A hereditary disease in which A deleterious mutant allele is replaced with A functional one.
  4. 4. TYPES OF GENE THERAPY Germ line gene therapy • In this case, germ cells, i.E., Sperm or eggs are modified by the introduction of functional genes, which are integrated into their genomes. •The change due to therapy would be heritable n would be passed on later generation. Somatic gene therapy • In this, therapeutic genes are transferred into the somatic cells of a patient. •Any modification and effects will be restricted to the individual patient only, & will not be inherited.
  5. 5. NEED OF GENE THERAPY • To overcome the disease. • Targeting the cells of diseased tissues. • Genes can be inserted into the cells of patients by direct and indirect routes, and the inserted genes can integrate into the chromosomes or remain extra chromosomal.
  6. 6. STRATEGIES OF GENE THERAPY •Gene augmentation therapy (GAT). • Targeted killing of specific cells. • Targeted mutation correction. • Targeted inhibition of gene expression.
  7. 7. GENE AUGMENTATION THERAPY • For diseases caused by loss of function of a gene, introducing extra copies of the normal gene may increase the amount of normal gene product to a level where the normal phenotype is restored. • Targeted to clinical disorders where the pathogenesis is reversible. • Applied to autosomal recessive disorders.
  8. 8. DISEASE CELLS GENE X NORMAL PHENOTYPE (increase in gene X product)
  9. 9. TARGETED KILLING OF SPECIFIC CELLS • Popular in cancer gene therapies. • Genes are directed to the target cells and then expressed to cause cell killing. • Direct cell killing – inserted gene are expressed to produce a lethal toxin or a gene encoding a prodrug is inserted, conferring susceptibility to killing by a subsequently administered drug.
  10. 10. DIRECT KILLING OF DISEASE CELLS DISEASE CELLS DISEASE CELLS CELLS KILLED BY EXPRESSED TOXIN CELLS KILLED BY DRUG PRODRUG GENE TOXIN GENE DRUG
  11. 11. TARGETED MUTATION CORRECTION • It can be done at different level: • At gene level, based on homologous recombination. • At rna transcript level, by using particular types • of therapeutic ribozymes.
  12. 12. TARGETED GENE MUTATION CORRECTION DISEASE CELLS (mutant gene X) NORMAL PHENOTYPE (genetic mutation corrected to restore functional gene) CORRECTED GENE GENE X mutant
  13. 13. TARGETED INHIBITION OF GENE EXPRESSION • If diseases cells display an inappropriate expression of a gene (in case of many cancers), a variety of different system can be used specifically to block the expression of a single gene at the DNA,RNA or PROTEIN levels.
  14. 14. Disease cells containing mutant or harmful gene Block of expression of pathogenic gene ANTISENSE GENE m m Inhibition AAAA N C
  15. 15. METHODS OF GENE TRANSFER • VIRAL METHODS • RETROVIRUS VECTORS • ADENOVIRUS VECTOR • HERPES SIMPLEX VECTORS • NON VIRAL METHOD • BY OLIGONUCLEOTIDES • BY LIPOSOME
  16. 16. Retroviruses are used for Gene Therapy LTR gag pol env LTR GENE X Packaging Signal LTR LTR 10 KB in size Gag Pol Env Packaging Cells = Vector Non-replication competent virus containing gene X, pseudotyped with selected envelope
  17. 17. Gene Transfer
  18. 18. BY RETROVIRAL VECTOR
  19. 19. VIRAL METHOD BY ADENOVIRUS VECTOR
  20. 20. BY LIPOSOMAL METHOD
  21. 21. GENE THERAPY FOR INHERITED DISORDERS • Recessively inherited disorders are conceptually the easiest inherited disorders to treat by gene therapy. • The first apparently successful gene therapy was initiated in 1990 for adenosine deaminase deficiency. • Cystic fibrosis. • Familial hypercholesterolemia (fh). • Gaucher’s diseases • For neoplastic disorders & infectious disease
  22. 22. GENE THERAPY SUCCESSES AND FAILURES SCID is often called "bubble boy disease". SCID became widely known during the 1970's and 80's, when the world learned of David Vetter, a boy with X-linked SCID, who lived for 12 years in a plastic, germ-free bubble. He died after a bone marrow transplant. A recessive disorder of a mutation in the adenosine deaminase (ADA) gene causes SCID. Gene therapy successfully replaced this gene in several ADA patients
  23. 23. GENE THERAPY TRIALS FOR INHERITED DISORDERS • DISORDER CELLS ALTERED GENE THERAPY STRATEGY ADA DEFICIENCY T-CELLS & HEMOPOITIC STEM CELLS EX VIVO GAT USING RECOMBIN-ANT RETROVIRUSES CONTAINING AN ADA GENE. CYSTIC FIBROSIS RESPIRATORY EPITHELIUM IN VIVO GAT USING RECOMBIN-ANT ADENOVIRUSES OR LIPOSOME-TO DELIVER THE CFTR GENE FAMILIAL LIVER CELLS EX VIVO GAT USING RETROVIRUS  HYPERCHOLESTEMIA TO DELIVER THE LDL RECEPTOR GENE. GAUCHER’S DISEASE HEMOPOITIC STEM CELLS EX VIVO GAT USING RETROVIRU-SES TO DELIVER THE GLUCO-CEREBROSIDASE GENE.
  24. 24. PROBLEMS AND ETHICS • SHORT-LIVED NATURE OF GENE THERAPY. • IMMUNE RESPONSE. • PROBLEMS WITH VIRAL VECTORS. •MULTIGENE DISORDERS. • CHANCE OF INDUCING A TUMOR.
  25. 25. CONCLUSIONS • It is believed that gene therapy will revolutionize the practice of medicine. This technique has the ability to cure many of the diseases that have effected our society for years i.E. People that have serious genetic diseases. • As scientist discover more genes & their functions, the potential for this treatment is limitless. As we know our fate is indeed in our gene.
  26. 26. REFRENCES • ASCADI G, DICKSON G, LOVE DR ET AL. NATURE, 352,815-818 • ANDERSON WF.(1985) J. MED. PHILOSOPH., 10,275-191. • ARBONES ML, AUSTIN HL, CAPON DJ, GREENBURG G. (1994) NATURE GENETICS,6,90-97. • BARINAGA M. (1993) SCIENCE, 262, 1512-1514. • BOCK LC, GRIFFIN LC, LATHAM JA, VERMAAS EH, TOOLE JJ. (1992) NATURE, 355, 564-566. • BORIS,LAWRIE K,TEMIN HM (1994) ANN. NY ACAD.SCI, 716, 59-71. • BRODY SL,CRYSTAL R.(1994)ANN. NY ACAD .SCI,716,90-103. • CECH T (1995) BIOTECHNOLOGY,13,323-326. • CHUBB JM,HOGAN ME,(1992)TRENDS BIOTECHNOL.,10,132-136.
  27. 27. • CULVER KW, BLAESE RM. (1994) TRENDS GENT., 10,174-178. CURIEL DT. (1994)ANN.NY ACAD.SCI,716,36-58. • ENGLAND SB,NICHOLSON LV, JOHNSON MA ET AL .(1990)NATURE,343,180-182. • GRLBA E,SMIT C. (199)TRENDS GENT.,10,139-144. GREEN LL, HARDY MC,MAYNARD-CURRIE CE ET AL.(1994)NATURE GENETICS,7,13-21. • GROSSMAN M, RAPER E,KOZARSKYR K ET AL.(1994) SCIENCE,232,1548- 1553. HODGOSON CP.(1995), • HAHN BH, SHAW GM, TAYLOR ME ET AL. (1986) SCIENCE, 232, 1548-1553.
  28. 28. SUSCRIBE US FOR MORE PRESENTATIONS….
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