Gene Therapy Slides ppt

Category: Education

Presentation Description

No description available.


Presentation Transcript


GENE THERAPY A Concise Presentation By Mr. Deepak Sarangi M.Pharm .( Ph.D ) 1


DEFINITION Gene therapy is the insertion ,alteration or removal of genes within an individual cells and biological tissues to treat disease. 2

PowerPoint Presentation:

What is gene therapy? Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or “knocking out,” a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. 3

PowerPoint Presentation:


How It Works:

How It Works A vector delivers the therapeutic gene into a patient’s target cell The target cells become infected with the viral vector The vector’s genetic material is inserted into the target cell Functional proteins are created from the therapeutic gene causing the cell to return to a normal state 5


APPROACHES FOR GENE THERAPY Gene modification: Replacement therapy Corrective gene therapy Gene transfer Gene transfer in specific cell lines Somatic gene therapy Germ line gene therapy Eugenic approach 6


GENE MODIFICATION REPLACEMENT THERAPY: In replacement therapy normal gene is inserted some where in the genome so that its product could replace that of defective gene. CORRECTIVE GENE THERAPY: In corrective gene therapy replacement of a mutant gene or a part of it with a normal sequence. 7


GENE TRANSFER Gene transfer can be used for improvement of a specific diseases. (eg: introducing growth hormone gene to increase the height ) 8


GENE TRANSFER TO SPESIFIC CELL LINES A.SOMATIC GENE THERAPY: Insertion of gene in to specific somatic cell. B.GERM LINE GENE THERAPY: Insertion of gene in to germ cells, i.e. into fertilised eggs. 9


EUGENIC APPROACH In eugenic aproch gene is inserted to improve complex traits of a person (eg: intellegence). 10

Gene Therapy Uses AIDS Virus to Fight AIDS :

Gene Therapy Uses AIDS Virus to Fight AIDS In the study, immune cells were removed from the patients' bodies, modified with a disabled AIDS virus known as a lentivirus, and then intravenously returned. The genetically altered cells disseminated anti-HIV material and prevented HIV from reproducing them. 11

Uses of gene therapy :

Uses of gene therapy Replace missing or defective genes; Deliver genes that speed the destruction of cancer cells; Supply genes that cause cancer cells to revert back to normal cells; Deliver bacterial or viral genes as a form of vaccination; Provide genes that promote the growth of new tissue; 12

Viruses are used as Delivery Tolls:

Viruses are used as Delivery Tolls Viruses are used as vectors to introduce the genetic material inside the bodies. These viruses are inactivated, they are not able to reproduce Adenoviruses Herpes viruses DNA tumor viruses Retroviruses RNA tumor viruses 13


VECTORS IN GENE THERAPY VIRUSES : All viruses bind to their Hosts and introduce their genetic material into the host cell as part of their replication cycle. RETROVIRUSES : Genetic material in retroviruses is in the form of RNA molecules, while the genetic material of their hosts is in the form of DNA. ADENOVIRUSES : These are viruses that carry their genetic material in the form of double stranded DNA. 14


HERPES SIMPLEX VIRUS : The herpes simplex virus is a human neurotropic virus. The wild type HSV-1 virus is able to infect neurons . 15

Physical Methods to Enhance Delivery:

Physical Methods to Enhance Delivery Electroporation Electroporation is a method that uses short pulses of voltage (75 kv to 150 kv) to carry DNA across the cell membrane . 16

PowerPoint Presentation:



MAJOR DEVELOPMENTS IN GENE THERAPY In 2007 and 2008, a man being treated by Gero Hutter was cured of HIV by repeated Hematopoietic stem cell transplantation . PROBLEMS: Some of the problems of gene therapy include: Short-lived nature of gene therapy Immune response. Problems with viral vectors. Multigene disorders. Chance of inducing a tumor. 18


conclusion Gene therapy is used in treatment of many genetic and acquired diseases. Gene therapy trials using retroviral vectors to treat X-Linked severe combined immunodeficiency (X-SCID) represent the most successful application of gene therapy to date. More research is going on through out the world. This field leaves a vast area of research for scientist, to explore new method of gene therapy to treat several dreadful diseases like AIDS. 19

PowerPoint Presentation:

REFERENCES: Kamimura K, Suda T, Zhang G, et al. (2011).  "Advances in Gene Delivery Systems" .  Pharm Med   Perez EE, Wang J, Miller JC,  et al.  (July 2008). "Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases".  Nat. Biotechnol .   Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD (September 2010). "Genome editing with engineered zinc finger nucleases".  Nat. Rev. Genet. Durai S, Mani M, Kandavelou K, Wu J, Porteus MH, Chandrasegaran S (2005).  "Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells" .  Nucleic Acids Res.   "The Process of Gene Therapy." Alternate Heals. 8 may 2006. Alternate Medicine, Web. 23 November 2009. Harwood, Adrian J. Protocols for Gene Analysis. 1st. 31. Totowa, New Jersey: Humana Press, 1994. 20

THANKS for viewing the ppt For more ppts on pharma related topics plz contact Or find me at following link :

21 THANKS for viewing the ppt For more ppts on pharma related topics plz contact Or find me at following link

authorStream Live Help