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Gene Therapy
 About Gene therapy
 What are the genes?
 How does gene therapy  work?
 Types of gene  therapy
 Viral gene delivery   systems
 Non-viral gene  delivery  systems
 Pro-drug activation  therapy
 Two-gene therapy
 Nucleic acid  therapeutic agents
 Antisense RNA  production
 Antisense  Oligonucleotides
 Nucleic acid  pharmaceuticals
 Ribozymes - as  therapeutic  agents
 Oligonucleotide  correction  of genetic
 History of events - gene  therapy
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Gene Therapy Introduction

Pro-drug Activation Therapy
 
  • Combination of HSV-thymidine kinase gene (HSV tk) and ganciclovir [GCV; 9-(1, 3-dihydroxy-2-propoxymethyl) guanine] has been used to eradicate proliferating tumor cells. 

  • Transfecting tumor cells in vivo with HSV tk gene under the control of an active promoter and after few days ganciclovir is administered.

  • HSV tk phosphorylates ganciclovir to form monophosphate  GCV. Triphosphate-GCV inhibits DNA polymerase and terminates DNA synthesis, causing death of tumor cell. 

  • Triphosphate-GCV can pass unmodified cells by cell to cell  contact and kill these cells as well. 1 HSV tk expressing tumor cell can kill up to 10 unmodified cells. This phenomenon is called “bystander effect”. 

  • When gene under certain conditions causes death of its own cell – suicide gene. 

  • Prodrug- Inactive form of therapeutic agent that is activated biologically after it is administered as part of a treatment.