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

What are genes?
  • Genes are present inside the chromosome, which are the basic physical and functional units of hereditary.
       

  • Genes are made up of specific sequences of bases and these gene bases, code for specific protein production as needed by the human body for normal metabolism.
       

  • Proteins are required for normal cellular function, proper functioning of proteins is required for production and maintaining of cellular structures.
      

  • When alterations in gene takes place the proteins which are coded by these gene are not able to function and causes genetic disorders.
      

  • Gene therapy is done to repair the defective genes responsible for disease. Gene therapy is done by: 

            1. Inserting the normal gene into the genome to replace the non 
                functional gene.
 
            2. By recombination: The abnormal gene is replaced by normal gene.
 
            3. By selective reverse mutation: The abnormal gene is repaired to 
               become normal and perform  its normal function.

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