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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

Antisense Oligonucleotides
  • Sequence specific effectiveness of chemically synthesized  antisense oligodeoxynucleotides on hybridization to an accessible site on target mRNA, resistance to degradation  by cellular nucleases, and are delivevered into cells.

  • Oligonucleotides with about 15-20 nucleotides have sufficient specificity to hybridize unique mRNA.

  • The most commonly used antisense oligonucleotides has a sulfur group in place of free oxygen of phosphodiester bond the RNA-DNA duplex activates endogenous enzyme  ribonuclease (RNase) H, which cleaves the mRNA component of hybrid molecule.

  • Clinical trials are undergoing using phosphorothioate  antisense oligonucleotides, against CMV, HIV.