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Mayo Clinic Jacksonville scientists show that specially created molecules can cross blood-brain barrier

t because not only is the molecule locking onto its intended target, it's also getting past the blood-brain barrier. This barrier blocks the action of many drugs on brain cells, making diseases that affect the brain often so difficult to treat.

To prove further that the PNA had reached the brain, Richelson's group developed an assay to measure the chemical's presence in brain cells.

The work is so unique that Mayo Clinic has applied for five U.S. and international patents on PNAs and the assay.

"It's absolutely mind boggling to see what these molecules did in animals," Richelson says. "What we've presented here goes totally against the grain. The medical literature tells us it shouldn't work. A huge skepticism for antisense molecules has developed because they seem to be unpredictable in that they're not binding to what you expect them to.

"The PNA field has been waiting for a major breakthrough in getting molecules into cells," Richelson says. "We're saying it's here. Inject PNAs and look for a biological response."

These findings have implications for treating many illnesses that are caused by overproduction of a protein normally found in the body and for treating diseases caused by foreign proteins invading the body (for example, viral infections). Scientists are just beginning to identify the specific proteins that are involved in certain diseases. But once the proteins are recognized, it will be possible to find the genes responsible for creating those proteins and develop antisense molecules to stop the proteins' function.

"We made a discovery that's totally unexpected, and it's taken us down a path we never dreamed of," Richelson says.


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Contact: Evelyn Tovar
tovar.evelyn@mayo.edu
904-953-7347
Mayo Clinic
1-Jul-1999


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