GENE THERAPY
• Definition of gene therapy:   “ Novel approach to treat,cure or ultimately    prevent a disease by changing the    expre...
Alright,tell mehow you want tochange…!                  Google images
Gene Therapy• Tell me how do you want to change       – Replacing a mutated gene that causes         disease with a health...
STEPS IN GENE THERAPY:2. Identification of the defective gene.3. Cloning of normal healthy gene.4. Identification of targe...
Cloned gene            (normal allele)   1 An RNA version of                                a normal human                ...
Cloned gene(normal allele)   1 An RNA version of                    a normal human                    gene is inserted    ...
2 Bone marrow cells                         are infected with                         the virus.                       3 V...
ore      e no m !  u ar nant!!Yo m i  Do                  Google images
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Gene therapy, brief overview

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  • Figure 12.10 One type of gene therapy procedure
  • Figure 12.10_1 One type of gene therapy procedure (part 1)
  • Figure 12.10_2 One type of gene therapy procedure (part 2)
  • Gene therapy, brief overview

    1. 1. GENE THERAPY
    2. 2. • Definition of gene therapy: “ Novel approach to treat,cure or ultimately prevent a disease by changing the expression of a person’s genes”Dr. Padmesh,V,Slideshare.net
    3. 3. Alright,tell mehow you want tochange…! Google images
    4. 4. Gene Therapy• Tell me how do you want to change – 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.Dr. Padmesh V, Slideshare.net
    5. 5. STEPS IN GENE THERAPY:2. Identification of the defective gene.3. Cloning of normal healthy gene.4. Identification of target cell / tissue / organ.5. Insertion of the normal functional gene into the host DNA.METHOD: Introduction of FUNCTIONAL GENES into appropriate cells Transferred gene (TRANSGENE) encodes & produces proteins The Proteins encoded by Transgene corrects the disorderDr. Padmesh V, Slideshare.net
    6. 6. Cloned gene (normal allele) 1 An RNA version of a normal human gene is inserted into a retrovirus. RNA genome of virus Retrovirus 2 Bone marrow cells are infected with the virus. 3 Viral DNA carrying the human gene inserts into the cell’s chromosome. Bone marrow cell from the patient4 The engineered cells are injected into the patient. Bone marrow
    7. 7. Cloned gene(normal allele) 1 An RNA version of a normal human gene is inserted into a retrovirus. RNA genome of virus Retrovirus
    8. 8. 2 Bone marrow cells are infected with the virus. 3 Viral DNA carrying the human gene inserts into the cell’s chromosome. Bone marrow cell from the patient4 The engineered cells are injected into the patient. Bone marrow
    9. 9. ore e no m ! u ar nant!!Yo m i Do Google images
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