anotherone121 t1_j0zwenj wrote
Reply to comment by bentlimerick in Gene therapy gel heals decades-old wounds in trial for blistering skin disease. Researchers find that a gel tested in patients with a life-threatening blistering skin disease helps wounds heal. The gel — the first topical gene therapy — awaits FDA approval. by Zee2A
>There's a line at the end of the paper "Longer and larger trials are warranted to determine the durability and side effects of B-VEC for this disease." that kind of misses the point.
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>Trials just don't last that long. Fifteen years is about as long as they come.
I suspect far shorter is fine. The concern around durability likely has to do with the vector used and the turnover of the tissue, transfected. Is the transgene being inserted as an epizome (doesn't multiply during cell replication... so gets diluted out over time) or is it integrating into the chromosome (--> safety concerns)? If it integrates, into what cell types, with what efficiency, and in what tissue layer, and how quickly do these turnover? In short, the clinical effect may be temporary.... quite temporary, depending on the technical details.
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