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Dr. Dietrich Stephan: New Drugs that Repair Broken Genes

Talking Biotech with Dr. Kevin Folta

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My Atonic Dystrophe

There are no effective therapies for my atonic distrophe at all, period. Most succumb to the disease in mid life. And we've shown that a single dose i v completelyres s normal splicing,. rescues the ability of transgentic animals to normally relax their muscles. We're drugging the double stranded arene that's toxic and still refractory to drugging using other genetic medicines. Our drug simply slips and invades that hairpin, re linearizes it and hysterically displaces all of those inappropriately sequestered proteins ot so they can now go do what they're supposed to do in the nucleus.

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