Gene Therapy Mechanisms


Like Stem Cells, Gene Therapy is a new area for clinical science that has a great potential for treating many disorders and diseases. Gene Therapy is basically like using DNA as the drug. Usually this is used to treat a mutation. Mutations are rare and can arise randomly; these can now be treated with the use of a Vector. The Vector would contain a therapeutic peptide (see earlier post regarding therapeutic peptides in Alzheimer’s disease). The diagram below outlines how a Vector works.


The Vector delivers recombinant viruses. The virus binds to its host and adds its genetic material, this is then encorporated into the host cell and passed on via cell replication. The viruses also spread through infection, this spreads as cells burst and infect other cells. The virus continues to reproduce itself and releases the DNA, this DNA is then used to create new viruses. This may sound like its using one problem to solve another however the use of viruses is used to deliver the DNA to the desired destination. This is known as a viral vector.  Another viral vector would be the use of retroviruses. This is becoming more common in Gene Therapy. They integrate into the genome without causing any instability. The retrovirus contains reverse transcriptase, this enzyme allows for the integration of the DNA into the genome. 

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