Tuesday, December 29, 2009

Will Zinc Finger "Scissors" Tools Help Achieve True Gene-Therapy?


In its "Science Tuesday" column today, The New York Times offers yet another provocative glimpse into what may be the future of medicine -- long hoped-for, but until this moment, largely unattainable: specific modifications to a patient's genetic structure. These precisely-targeted changes would be used to fight disease, to boost immunity, or (in perhaps the most ambitious version) to render the patient's DNA impervious to attack from the HIV virus, for example.

[This very optimistic view stands in stark contrast to rather down-beat pieces I profiled in the NYT, earlier, on genetic science to treat disease.]

Do go read it all, but here is a snippet:

. . . .Zinc fingers are essential components of proteins used by living cells to turn genes on and off. Their name derives from the atom of zinc that holds two loops of protein together to form a “finger.” Because the fingers recognize specific sequences of DNA, they guide the control proteins to the exact site where their target gene begins.

After many years of development, biologists have learned how to modify nature’s DNA recognition system into a general system for manipulating genes. Each natural zinc finger recognizes a set of three letters, or bases, on the DNA molecule. By stringing three or four fingers together, researchers can generate artificial proteins that match a particular site.

The new system has been developed by a small biotech company, Sangamo BioSciences of Richmond, Calif., and, to some degree separately, by academic researchers who belong to the Zinc Finger Consortium. . . .

Again, if this pans out, the implications for old-line pill makers could hardly be more profound. Bio-science programs at New Merck (and Pfizer, Sanofi, Amgen and Novartis) seek to be ready, and in a position to capitalize -- should this zinc scissoring "tool-belt" emerge as a feasible way to handle the gene sequence, with precision. To be clear, though, the whole idea of treating at the genetic level, rather than along old-line chemical absorption pathways, is as different from -- and revolutionarily-beyond -- the "old" medicines, as (in transportation) the space-shuttle is. . . to an ox-cart on a rutted dirt path.

[Note: My image, at above-right, is highly modified, but the original 3D render was from a public domain wiki-image bank. And so, I declare it too as in the public domain, with my modifications.]

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