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Minor Update to Cullen/Bloom/Editas Collaboration


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As many of us know, Dr. Cullen, Dr. Bloom and Editas are working together to cure HSV-1 in mice.  I got some information from Dr. Cullen and thought I would share regarding where they are right now.  It looks like in a couple of weeks they will know whether or not the treatment worked in mice.  I'm not sure if they will release the data prior to publishing it in a journal, so it may be a few months before the general public gets wind of the data.

As you know, we are working with Editas and Bloom to cure HSV-1 in mice using AAV vectors designed to deliver Cas9 of  Staph. aureus origin to the TG. Working with Editas, we designed a wide range of guide RNAs targeting various genes in HSV-1, including ICP4 and ICP0, and tested these for efficacy in tissue culture, We narrowed the guides down to the 5 most effective and then engineered AAV to express one or two of these. The AAV was packaged and is now being tested in mice. Things went a little slower that we had hoped but, depending on how fast Bloom gets these experiments done, we hope to have data addressing the cure rate in mice in a few weeks.
Actually, we find that cleaved episomal DNA is destroyed, so it doesn’t seem to make much difference where you cleave, as long as cleavage is efficient. We’ll see if this holds true in vivo.

Interesting that it doesn't matter where the protein is cleaved, as long as it is cleaved the DNA is destroyed.  Data from the mice will confirm whether this is accurate in live specimens (let's hope it is).

Edited by Evaluate
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Not yet. There's no timeline for when the data would be released. As I mentioned they might not say anything until published in a journal, so it might be months. Then again, Cullen might give an update early. Time will tell.

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Would be nice if they posted here from time to time like Chip Clark from Genocea. Also, HSV1 and HSV2 contain many similar proteins. Could the CRISPR they designed for HSV1 also destroy HSV2

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5 hours ago, mcmich said:

Would be nice if they posted here from time to time like Chip Clark from Genocea. Also, HSV1 and HSV2 contain many similar proteins. Could the CRISPR they designed for HSV1 also destroy HSV2

It would be nice, but the fact that Cullen posts regular updates on a website is good enough for me.

I'm not a scientist, but I think it depends on how specific the guide RNAs are.  Right now it seems they are doing their best to prevent off targeting and to hit their mark, which means working hard to be specific about the guide RNA.  If their concept works for HSV-1 and makes an effective cleavage and disables the virus, it in theory should work exactly the same with HSV-2.  Then, creating a guide RNA for HSV-2 should not be difficult after success with HSV-1.

Start small, show proof of concept, then expand once the FDA is happy.

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I'm sure if there is news Cullen would post it to his site.  Like I said, they may wait to publish it in a journal article first so we may not end up hearing anything until that happens.

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