Shrinking Genes - MDA Research for DMD Treatment

[wasteofo2]

this is an exerpt from this article: http://www.mdausa.org/publications/Quest/q92resup.cfm#mdasgenetherapy


"Meanwhile, MDA is also laying the groundwork for a clinical trial of AAV-based gene therapy for Duchenne muscular dystrophy (DMD). DMD results from any of a number of mutations in a large gene for the muscle protein known as dystrophin. The full-size gene can't be packaged inside an AAV vector.

To overcome this problem, MDA grantee Jeffrey Chamberlain, a molecular biologist at the University of Washington in Seattle, has developed highly miniaturized dystrophin genes that lead to the production of small proteins. Studies using these microdystrophin genes, which were published in the March issue of Nature Medicine, showed extremely promising results in correcting muscle abnormalities in dystrophin-deficient mice."

I'm shocked. I had no idea that genes, or anything of that sort could be shrunken at all, and the way which they say it makes it sound like a relatively easy thing to do. Has anyone ever heard of a process able to shrink down genes, or any other parts of cells? Would it be at all possible to shrink organelles, or perhaps make a virus which would alter the genes of a cell to make everything smaller as a whole when it divides?

 

[Ambitwistor]

They didn't literally shrink any genes. What they did was make a new gene that contained only pieces of the original gene, after they found that only certain parts of the gene produced the protein functions they wanted. This has nothing to do with making cells, or parts of cells, smaller.

For a few more details, see here:

http://www.pitt.edu/utimes/issues/33/010111/research.html

 

[rapa-nui]

So... if I understand correctly basically they got rid of all the introns, enhancer sites that weren't strictly needed, proteins that weren't strictly needed, and a bunch of other redundant junk?

Pretty clever actually.