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Biotech / Medical : ARIAD Pharmaceuticals
ARIA 23.990.0%Feb 17 4:00 PM EST
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To: Pseudo Biologist who wrote (972)2/3/2000 8:20:00 PM
From: Pseudo Biologist  Read Replies (2) of 4474
 
Quick description of the "RAPID" paper:

Make a vector that codes for the following components:

a signal sequence+a "CAD"+furin cleavage sequence+"insulin"

"CAD" is a "conditional aggregation domain," actually four tandem repeats of a mutated version of FKBP12. As the name suggests, the protein coded by this tends to aggregate, and keeps the whole fusion construct "arrested" in the ER. Result: create a "depo" of insulin inside the cell.

Adding a small molecule "antidimerizer" blocks the CAD aggregates and this allows furin, an enzyme that already exists in the cells, to cut the "insulin" and let it out of the ER and into action. The details of how the CAD and the small molecule interact with each follow very closely from what dimerizers do in ARGENT. This is the main logical connection to ARGENT proper.

("insulin" - I use quotes because the situation is a bit more complex than described, due to the multi-chain nature of this protein. "insulin" of course may be replaced by any other protein for which this "burst" expression may make sense)

Therapeutic application *may* be easier than with ARGENT in part because a single gene, say in an AAV vector, can be used to deliver the whole thing.

Hope this makes some sense, if not just shout and we will try again.

PB
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