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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

Nucleic acid Therapeutic agents
  • Therapeutic systems using nucleotide sequences are devised to treat human disorders.

  • Small, single strand nucleotide sequences  (oligonucleotides) could hybridize to a specific gene or mRNA and reduce transcription or translation, lowering the amount of protein synthesized.

  • An oligonucleotides designed to bind to a gene and block transcription is called antigen oligonucleotides and one that base pairs with specific mRNA is an antisense oligonucleotides.

  • Binding of oligonucleotides to transcription factor responsible for expression of specific gene. Double stranded oligonucleotides that attach to DNA binding proteins could prevent activation of transcription of specific genes.

  • Synthetic DNA molecules that bind to proteins and prevent them from functioning can be created.

  • Ribozymes are natural RNA sequences that bind and cleave  specific RNA molecules. These could be engineered to  target an mRNA and decrease the amount of proteon.