INTRODUCTION ‡ Gene therapy is the insertion. ‡ It is a technique for correcting defective genes that are responsible for disease development. alteration. ‡ The most common form of gene therapy involves the insertion of functional genes into an unspecified genomic location in order to replace a mutated gene. focusing on diseases caused by singlegene defects. or removal of genes within an individual's cells and biological tissues to treat disease. . ‡ Scientists have taken the logical step of trying to introduce genes directly into human cells. ‡ other forms involve directly correcting the mutation or modifying normal gene that enables a viral infection.

‡ most gene therapy studies are aimed at cancer and hereditary diseases linked to a genetic defect. muscular dystrophy and sickle cell anemia. ‡ cystic fibrosis.. . haemophilia.CONTD. ‡ this has proven more difficult than modifying bacteria. primarily because of the problems involved in carrying large sections of DNA and delivering them to the correct site on the gene.

genetically-mediated therapy. ‡ The most common form of genetic engineering involves the insertion of a functional gene at an unspecified location in the host genome. ‡ This is accomplished by isolating and copying the gene of interest. but is a related. generating a construct containing all the genetic elements for correct expression.CONTD ‡ Antisense therapy is not strictly a form of gene therapy. . and then inserting this construct into a random location in the host organism.

‡ gene-knockout mediated gene therapy is the knockout of the human CCR5 gene in T-cells in order to control HIV infection.OTHER FORMS OF GENETIC PROCESS ‡ gene targeting ‡ knocking out specific genes via engineered nucleases. . ‡ zinc finger nucleases ‡ engineered I-CreI homing endonucleases. ‡ nucleases generated from TAL effectors.

Germ cells. Germ line gene therapy ‡ In the case of germ line gene therapy.e. i.TYPES OF GENE THEORY 1.. ‡ the change due to therapy would be heritable and would be passed on to later generations. . sperm or eggs. are modified by the introduction of functional genes.

and will not be inherited by the patient's offspring or later generations. ‡ Any modifications and effects will be restricted to the individual patient only. the therapeutic genes are transferred into the somatic cells of a patient. .SOMATIC GENE THERAPY ‡ n the case of somatic gene therapy.

1.1 RETRO VIRUS 1.5 Cis and trans-acting elements .VECTORS IN GENE THERAPY 1. VIRUS 1.3 Envelope protein pseudotyping of viral vectors.4 Replication-Competent Vectors 1.2 ADENO VIRUS 1.

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. .

. ‡ In 1972 Friedmann and Roblin authored a paper in Science titled "Gene therapy for human genetic disease.MAJOR DEVELOPMENTS ‡ Gene therapy is being studied in clinical trials (research studies with people) for many different types of cancer and for other diseases.

1990.CONTD ‡ The first approved gene therapy case in the United States took place on September 14. Los Angeles research team inserted genes into the brain using liposomes coated in a polymer called polyethylene glycol. at the National Institute of Health. It was performed on a four year old girl named Ashanti DeSilva ‡ In 2003 a University of California. .

‡ 3. .Introducing a new gene into the body to help fight a disease.ACHIEVEMENTS ‡ Replacing a mutated gene that causes disease with a healthy copy of the gene. ‡ Inativating or knocking out. a mutated gene that is functioning improperly.

USES OF GENE THERAPY ‡ Replace missing or defective genes. ‡ Deliver bacterial or viral genes as a form of vaccination. ‡ Deliver genes that speed the destruction of cancer cells. . ‡ Supply genes that cause cancer cells to revert back to normal cells.

‡ An abnormal gene could be swapped for a normal gene through homologous recombination. This approach is most common.GOALS ‡ A normal gene may be inserted into a nonspecific location within the genome to replace a non-functional gene. ‡ The abnormal gene could be repaired through selective reverse mutation. which returns the gene to its normal function. .

CONTD ‡ The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered. .