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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

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Non-viral Gene delivery systems
 
  • All viral vectors which are used to transfer genes induce an immunological response to some degree & may have safety risks (such as insertional mutagenesis & toxicity  problems).

  • Furthermore their capacity is limited & large scale  production may be difficult to achieve.

  • Non-viral methods of DNA transfer require only a small number of proteins, have a virtually infinite capacity, have no infectious or mutagenic capability & large scale production is possible using pharmaceutical techniques. 

  • Viral vectors are costly to maintain 

  • Has limited cloning capacity 

  • Viral proteins may induce inflammatory response. 

  • Limitations:
      
        1. Frequency of transfection is often too low to create a
            therapeutic effect.
      
        2. Duration of therapeutic gene ex-pression is too brief to 
            provide an effective treatment. 
      

  • There are three methods of non-viral DNA transfer.  namely: 

         1. Naked DNA
      
         2. Plasmid liposomes complex
      
         3. Molecular conjugates 

    Naked DNA:

  • Naked DNA is directly injected inside the target tissue. 

  • In this procedure the pure DNA constructs are directly inserted into cells of target tissues. 

  • Therapeutic genes delivered are expressed in targeted tissues and gene products are released into circulatory system. 

  • Secretion of therapeutic protein into circulatory system should facilitate into target tissue.

  • DNA construct (genetic DNA) is surrounded by artificial  lipid layers to form a lipid sphere with an aqueous core which facilitates passage of DNA through the cell membrane. 
      

  • This method is a simple procedure and has shown good expression in target genes.

  • This technique is used for DNA vaccine productions. 

  • This technique does not produce immunity against the agents; it is relatively a cheap technique and can be used for multiple deliver. 

  • By transferring DNA, it is able to immunize with two serologically distinct strains.   

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