UCL INSTITUTE OF OPHTHALMOLOGY
DIVISION OF MOLECULAR THERAPY
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Gene Therapy
Virus particle used for gene therapy
Virus particle used for gene therapy

What is gene therapy?
The concept of gene therapy is elegant in its simplicity: if cells cannot function because a gene they require is mutated, they should be fine if we can replace the mutated gene with a correct version.

With modern technology it is fairly easy to isolate the correct gene, but how do we get it into the cells that need it? Nature provides us with the answer: viruses. Normally, viruses enter their own genes into the host cell in order to take over control of the cell and force it to produce more viruses. We can use these viruses for gene therapy by removing the virus genes from the virus particle, thereby making the virus harmless and replacing it with the gene we want to put into the cells. The virus will deliver this gene to the cell and instead of taking control of the cell, it will correct the defect in that cell.

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

This page last modified 18 December, 2012 by xxx


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