Gene Therapy

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Gene Therapy
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Antisense Technologies

• antisense oligonucleotides(ODNs)
• Ribozymes
• DNAzymes
• RNA interference(RNAi).

Part of Gene Therapy
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Gene Therapy... ----idea is to replace defective
genes 
Gene therapy is a technique for correcting
defective genes responsible for disease
development.
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Several approaches for correcting faulty genes

1. A normal gene may be inserted into a nonspecific
   location within the genome to replace a
   nonfunctional gene.
2. An abnormal gene could be swapped for a normal
   gene through homologous recombination.
3. The abnormal gene could be repaired through
   selective reverse mutation, which returns the
   gene to its normal function.
4. The regulation (the degree to which a gene is
   turned on or off) of a particular gene could be
   altered.

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Deliver the therapeutic gene

• Some of the different types of viruses used as
  gene therapy vectors
• Retroviruses
• Adenoviruses
• Adeno-associated viruses
• Herpes simplex viruses

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Deliver the therapeutic gene

• Adenoviruses

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Challenge

• Efficient delivery
• Enhanced stability
• Minimization of off-target effects
• Identification of sensitive sites in the target
  RNAs

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Status of gene therapy

• The Food and Drug Administration (FDA) has not
  yet approved any human gene therapy product for
  sale.

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Gene Therapy in the clinic Trials Wordldwide
66 Phase I 21 Phase I-II 11 Phase II 0.8
Phase II-III 0.7 Phase III

• in spite of 13 year- research only less than 1
  of the trials has reached phase III

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For more details

• Genetic Modification Clinical Research
  Information System (GeMCRIS)
• http//www.gemcris.od.nih.gov/

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Thanks!!

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