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

How does gene therapy work?
  • Genes are composed of Deoxyribonucleic acid – DNA. DNA is composed of genetic information, by which they produce the specific proteins which are the building blocks of the body. 
      

  • Gene damage or missing gene may lead to inadequate or wrong protein production leading to disease condition.
      

  • In gene therapy, a normal gene is inserted into the genome to replace an abnormal disease causing gene.
      

  • A carrier molecule “vector” can be used to deliver the therapeutic gene into the patients target cells.
      

  • Viruses are microorganisms which infect humans and multiply in the genome of the human body.
      

  • Scientists have tried to take advantage of this multiplication capacity of the viruses to transfer the required genes into the human body for treatment purposes.
      

  • The virus is manipulated by the scientists and the pathogenic disease causing genes in the virus are removed and the therapeutic genes are inserted.
      

  • The virus vector then unloads its genetic material containing the therapeutic human gene into the target cell (human host).
      

  • The treated human produce the normal proteins due to the transfer of the therapeutic genes. 
       

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