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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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Types of gene therapy
  • The transduced cells are grown in culture, collected  in large amounts, and then introduced into the      patient at various intervals; with the hope that the  cells will be maintained and the disease that is being treated will be corrected.   

  • Patients with genetic diseases that respond to bone  marrow transplantation are treated by ex vivo gene therapy.   

  • Bone marrow transplantation acts as a therapeutic    procedure for different genetic diseases, bone marrow  contains totipotent embryonic stem cells at a    frequency of 10-4 to 10-5. These embryonic cells can     divide and differentiate into various important cell    types, including B and T lymphocytes, macrophages, Red blood cells, platelets and bone cells.   

  • Ex vivo gene therapy is a procedure in which genetically engineered totipotent stem cells are transplanted into a patient, where they would provide the missing cell type or gene product.  

In-vivo Gene therapy:

  • Gene therapy involves direct delivery of a corrected  gene into the cells of a particular tissue of patient.  

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