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Vertex Pharmaceuticals Incorporated Message Board

  • verityvoila verityvoila May 11, 2012 8:07 AM Flag

    Hypothesis II: f508d heterozygotes will benefit as much, if not more, from 809/770 Combo

    Hypothesis II: f508d Heterzygotes will see as much, if not more success, from 809/770 Combo:

    1. My initial thought for this hypothesis was that with heterzygotes, the non-f508d mutation will likely have more folding, sequencing, or trafficking potential than f508d, and thus will benefit more from 770 alone. I still think this.
    2. But recent trolling discovered this additional theory–transcomplementation: In short, there are “many laboratory studies demonstrating that when two different CFTR mutant proteins are co-expressed, mutant CFTR proteins move to the cell surface that would not traffic when expressed alone because of transcomplementation between the mutant proteins.” Full explanation available here:

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    • I am learning a lot from your hypotheses. Transcomplementation is a reasonable theory. It sounds as if it is related to what molecular geneticist call [rescue] of a defect in a gene by a different defect in the other location. I'll think about what you are saying over the weekend.

      • 1 Reply to thirdmeinvestor
      • Looking forward to your insights. Also, it is possible that with homozygotes that "rescue" occurs or transcomplementation with the remaining mutant protein? In other words, with homozygotes, the 809 and/or 770 fix part of the defect, but not all, and that alters the universe of mutants and their various relationship so the body then readjusts and those new mutants/fixed mutants react with each other differently so have better improvement than what the drugs cause? Wonder if that explains the unexpected stellar results. . . . I also think part of it is explained by the fact that you have proteins on two allelles fixed and not merely the one 551f allelle.

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