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Biotech / Medical : Gene therapy -- Ignore unavailable to you. Want to Upgrade?


To: tuck who wrote (298)2/6/2004 1:35:03 PM
From: JMarcus  Respond to of 319
 
<<This looks like a problem.>> I'm not sure that this as much of a problem needing a solution as it is an explanation of why AAV vectors do not work even better than they do. The effect of DNA-PK on the AAV vectors appears to be a factor that decreases the integration of the payload, but the preclinical and clinical trials performed to date show that this limiting factor is not preventing the vectors from achieving therapeutic efficacy. Having identified the role of DNA-PK, we now have a target for optimizing the effectiveness of the AAV vectors. But your pessimism about being able to successfully attack that target seems justified.

Marc