Single-digit Factor IX levels would not be normal; the patient with severe hemophilia would then have mild hemophilia, still a great improvement. These patients (mild) typically only manifest symptoms with trauma or surgery. At a cost of 1/2 to 1 million dollars a year for conventional prophylactic therapy the economic incentives for development would be huge. I assume the next step will be dog testing, as there is a hemophilia model there. Then?? Any knowledge of the pipeline time-line to human trials and ...successful deployment? 5 years? 10? The two big questions in my mind are whether the ZFN technology can eliminate the mutation issue and subsequently immune response issues (inhibitor development).
How does this translate to Cystic fibrosis? I'm not sure what level of corrected CFTR is necessary for significant disease/symptom abatement. Not sure if it is linear, or somewhat like hemophilia in the need for less than full conversion of target genes. Also there is an issue with the larger size of the CF gene involved as opposed to the FIX gene which is about 1/3 the size, I believe. At what point is gene size a detriment to ZFN deployment and function?
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