Background on this question: CRISPR-Cas is a naturally occurring process in bacteria that is used to adapt to viral attacks. We've coopted the system for use in mammals. As far as I know a few labs in this space are…
> I would deal with this by deciding how many cells I want to kill each day Yes, this is an approach. This starts to exceed my understanding of the problems the teams are facing - but there are concerns about the…
> How much overall has this costed you? For this project, seed capital low six figures. I am collaborating with family and friends, non-profits, and using doubling mechanisms available to me to at work to fund the very…
> I don't think that more than sloppy accuracy is acceptable for any gene therapy Valid critiques of Cas12a2 must acknowledge the mechanistic differences between Cas9 and Cas12a2. There is no research to suggest Cas12a2…
> Are there any plans to move to in vivo? Yes! That's the next step. There wasn't a mouse model for my variant so they're building that, too. But in vivo testing should be underway this calendar year.
Done! Wrote a bit more in a comment above. Thank you for the interest.
I have now :) I replied to a sibling comment. Thank you for the nudge to share.
Thank you. I want to make sure you see the comment above - I think it was your comment that nudged me into writing it :) Happy to answer any other questions, here or over email (in my user profile). > other folks…
> Healthy cells will also get these nanoparticles, but without the triggering DNA sequence, the mRNA payload will remain inert and eventually will be degraded. This is my understanding as well.
> So how does Cas12a2 mitigate off-target effects? Others in this thread may be able to give a better analogy, but I'll try: Cas9 is like open heart surgery on millions of cells all at once. We know the specific outcome…
I haven't written about it publicly, but I can elaborate here. I don't mind answering further questions about it even if you believe they'd make me uncomfortable - they won't. I've come to terms with what's happening to…
There are two major problems, delivery is one of them. Collateral damage of mass cell destruction leading to systemic inflammation is the other. The approach I'm reviewing now uses lipid nanoparticles (LNPs) for…
You're correct about CRISPR Cas9. The off-target affects are difficult to manage. The paper describes Cas12a2. This is a different mechanism with discovery origins in - of all things - agriculture. It does not attempt…
The cost of cartridges are comparable to optical disks. However, LTO drives are non-trivial in terms of both mechanical complexity and cost. I can toss an optical tray into the recycle bin without batting an eye. Not…
> I don't think the evidence we have is reliable enough to call someone "wildly misinformed" or "intentionally misleading", though. Perhaps not. However, my original challenge to rayiner's comment has produced far more…
You would think they'd have lifetime appointments as other federal courts do, but it's important to note FISC (and its review process) isn't the final stop on some Orwellian and diabolical train. It is very easy to get…
The argument for a persons "right to die" may hold up to some degree. However, I believe the spirit of that sort of protection applies to those who are terminally ill. The idea being able to preserve a persons dignity…
Slightly off-topic. Years ago when I was working at a small ISP in Columbia, SC I had lunch with Austin Meyer. He came in to upload the latest release of X-Plane to his FTP servers. We went across the street to get some…
It isn't a problem if you're speaking specifically about Cloudant. They co-locate clusters next to your app as best they can (as Mike has mentioned elsewhere). That makes the network you're running on critical, however.…
You cannot renounce your citizenship while in the country. The process requires you leave the US, enter an embassy or consulate and declare your intention to renounce. File some paperwork and you're no longer a citizen.…
Background on this question: CRISPR-Cas is a naturally occurring process in bacteria that is used to adapt to viral attacks. We've coopted the system for use in mammals. As far as I know a few labs in this space are…
> I would deal with this by deciding how many cells I want to kill each day Yes, this is an approach. This starts to exceed my understanding of the problems the teams are facing - but there are concerns about the…
> How much overall has this costed you? For this project, seed capital low six figures. I am collaborating with family and friends, non-profits, and using doubling mechanisms available to me to at work to fund the very…
> I don't think that more than sloppy accuracy is acceptable for any gene therapy Valid critiques of Cas12a2 must acknowledge the mechanistic differences between Cas9 and Cas12a2. There is no research to suggest Cas12a2…
> Are there any plans to move to in vivo? Yes! That's the next step. There wasn't a mouse model for my variant so they're building that, too. But in vivo testing should be underway this calendar year.
Done! Wrote a bit more in a comment above. Thank you for the interest.
I have now :) I replied to a sibling comment. Thank you for the nudge to share.
Thank you. I want to make sure you see the comment above - I think it was your comment that nudged me into writing it :) Happy to answer any other questions, here or over email (in my user profile). > other folks…
> Healthy cells will also get these nanoparticles, but without the triggering DNA sequence, the mRNA payload will remain inert and eventually will be degraded. This is my understanding as well.
> So how does Cas12a2 mitigate off-target effects? Others in this thread may be able to give a better analogy, but I'll try: Cas9 is like open heart surgery on millions of cells all at once. We know the specific outcome…
I haven't written about it publicly, but I can elaborate here. I don't mind answering further questions about it even if you believe they'd make me uncomfortable - they won't. I've come to terms with what's happening to…
There are two major problems, delivery is one of them. Collateral damage of mass cell destruction leading to systemic inflammation is the other. The approach I'm reviewing now uses lipid nanoparticles (LNPs) for…
You're correct about CRISPR Cas9. The off-target affects are difficult to manage. The paper describes Cas12a2. This is a different mechanism with discovery origins in - of all things - agriculture. It does not attempt…
The cost of cartridges are comparable to optical disks. However, LTO drives are non-trivial in terms of both mechanical complexity and cost. I can toss an optical tray into the recycle bin without batting an eye. Not…
> I don't think the evidence we have is reliable enough to call someone "wildly misinformed" or "intentionally misleading", though. Perhaps not. However, my original challenge to rayiner's comment has produced far more…
You would think they'd have lifetime appointments as other federal courts do, but it's important to note FISC (and its review process) isn't the final stop on some Orwellian and diabolical train. It is very easy to get…
The argument for a persons "right to die" may hold up to some degree. However, I believe the spirit of that sort of protection applies to those who are terminally ill. The idea being able to preserve a persons dignity…
Slightly off-topic. Years ago when I was working at a small ISP in Columbia, SC I had lunch with Austin Meyer. He came in to upload the latest release of X-Plane to his FTP servers. We went across the street to get some…
It isn't a problem if you're speaking specifically about Cloudant. They co-locate clusters next to your app as best they can (as Mike has mentioned elsewhere). That makes the network you're running on critical, however.…
You cannot renounce your citizenship while in the country. The process requires you leave the US, enter an embassy or consulate and declare your intention to renounce. File some paperwork and you're no longer a citizen.…