A Big Step Up For Gene Therapy: Permanence

This news is most interesting:

"To date gene therapy has relied upon vectors that randomly insert genes into the cell's genome," explains Savio L. C. Woo, PhD, Professor and Chairman of Gene and Cell Medicine at Mount Sinai School of Medicine and corresponding author on the study. "The technique we developed identifies a specific sequence which only occurs in a few places in the mammalian genome. These sequences occur between genes so there is no danger of the insertion of the gene damaging existing genes in the cell.

"Because the genes are inserted permanently, a few applications would suffice to permanently correct a disease."

The key word here is "permanently." Genes inserted using existing methodologies don't stick around for long: gene therapy under those conditions is more analagous to courses of medication in that the patient's biochemistry is altered for a limited period of time only. Now, however, permanent correction is a possibility. That's a big step forward in the potential quality and effectiveness of gene therapy - it opens the door to comparatively low cost therapies for any condition that could be cured by adding a new gene or an additional working copy of an existing gene. We're one step closer to being able to reconfigure an adult human genome for therapeutic benefit - what interesting times we live in!

UPDATE: You'll find a discussion thread on this advance over at the Immortality Institute forum.

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Comments

Gene therapy is going to be huge when we can make permanent changes.

Posted by: AA2 at October 14th, 2005 2:08 PM
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