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jshaffer85

10/01/11 3:04 PM

#124 RE: Teedlum #122

It is not a zero-sum game. Other patients had both CCR5 copies disrupted but the heterozygote had larger percentages given that he had only one copy of the CCR5 gene to begin before the procedure. One non-heterozygote had a 2 log reduction in viral load during the 12 week treatment interruption.

The basic proof of concept has been demonstrated and the cells clearly have an antiviral effect. It is now a matter of optimization to get the percentage of biallelically modified cells high enough to reduce the virus to undetectable levels.