Posted by: menotmd | June 9, 2010

Gene Therapy #sciboot10

Gene Therapy – Are we there yet?” Speaker: Guangping Gao, Gene Therapy Center, UMass Medical School

Gene Therapy is the next topic of discussion:

We were given an overview of gene therapy. The speaker defined gene therapy as the treatment, cure or prevention of disease by modification of the expressions of one’s gene.

So what does this mean?

It means that if you have a disease or a disorder that is of genetic origin, sometimes researchers will remove a part of the portion of the gene (typically that has a single gene defect like sickle cell and cystic fibrosis) that isn’t working, and replace with a gene that does. It isn’t that easy and sometimes it does work, obviously there are risks that are associated with receiving gene therapy. So why invest in it? Because if we can figure out the glitches, we can repair gene mutations (sickle cell anemia), restore missing functions (diabetes) and/or silence genes (cystic fibrosis/cancer).

So what’s the problem?

The problem is the delivery vehicle (virus/vector). How are we going to get the correct gene sequence to the target cell?

There are two ways that gene therapy is accomplished:

Ex Vivo – which is the typically method. Where the part of the gene is removed from the body (through body or biopsy), cultured (meaning enough is made so that researchers can continually test it) and then tested.
In Vivo – where the gene therapy is directly applied into your body and hopefully reaches the right cells and implements the information changes (think invasion of the body snatches or maybe, Spiderman is a better example! The spider bit spider man and rearranged his genetic information so that he got superpowers).

Currently researchers are seeking a vector that doesn’t produce an immunological effect, like what happens with folks who get transplants and end up with graft versus host disease . Adeno-Associated Virus seems to be the choice of some scientists because it doesn’t produce an immune response, isn’t pathogenic and there’s plenty of them.

So I am going to leave you with this video, because my head hurts from thinking to much (it’s genetic).


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