Back in 2011, there was an announcement from MIT about a new approach to a broad-spectrum antiviral that appeared to work.[1] This goes way beyond an AIDS-specific cure.
But it was at MIT Lincoln Labs, which doesn't usually do bio. So the researcher moved to Draper Labs, but didn't get much funding. Then that funding ran out. Now the guy behind this is trying to get funding on Indiegogo.[3] The problem seems to be that it's too far along for small-scale YC-sized funding, but not far enough along to sell to Big Pharma. The guy behind it clearly doesn't know how to get funded. He has a web site [4] and keeps trying for crowd funding.<p>Some VC needs to talk to this guy. This might or might not work, but the upside is good and the costs aren't that high.<p>[1] <a href="https://www.ll.mit.edu/news/DRACO.html" rel="nofollow">https://www.ll.mit.edu/news/DRACO.html</a>
[2] <a href="http://www.businessinsider.com/todd-rider-draco-crowdfunding-broad-spectrum-antiviral-2015-12" rel="nofollow">http://www.businessinsider.com/todd-rider-draco-crowdfunding...</a>
[3] <a href="https://www.indiegogo.com/projects/dracos-may-be-effective-against-all-viruses#/" rel="nofollow">https://www.indiegogo.com/projects/dracos-may-be-effective-a...</a>
[4] <a href="https://riderinstitute.org/" rel="nofollow">https://riderinstitute.org/</a>
For anyone unfamiliar with CRISPR, I strongly recommend this quick introduction video from Kurzgesagt on YouTube:<p><a href="https://www.youtube.com/watch?v=jAhjPd4uNFY" rel="nofollow">https://www.youtube.com/watch?v=jAhjPd4uNFY</a>
Wow, I had not connected the idea of using CRISPR genome editing with targeting virus dna segments. If I understand the technique correctly (and I am not a biologist for sure!) they used an adeno virus modified with a specific Cas9 setup to elide HIV DNA from cells it infected (normally I associate viruses with <i>adding</i> DNA rather than removing it :-)<p>Is that even close to a correct interpretation? It sounds like the technique could be used for pretty much any virus dna you wanted to target.
It is worth noting that the real challenge in gene therapy at this time is not editing the genes, but in getting sufficient coverage of edited cells (and especially progenitors and stem cells) in an adult individual to achieve the therapeutic goal and make it last.<p>(Most studies of genetic alterations in the broader sense have bypassed this challenge by working with animal lineages, or animals in which the editing happens in the earliest stages of development, when there are very few cells needing to be changed. The alteration then propagates during embryonic and later development).<p>There have been a number of very promising studies in the past year or two with regard to gene therapies to apply to adults, such as animal studies that demonstrated a cure for an inherited muscular dystrophy, but in the bigger picture, comprehensive coverage of tissues and cells is still something that the research community is in the midst of getting to grips with.
If (like me) you don't have a good working understanding of CRISPR, check out this fantastic episode of Radiolab: <a href="http://www.radiolab.org/story/antibodies-part-1-crispr/" rel="nofollow">http://www.radiolab.org/story/antibodies-part-1-crispr/</a>
Full article is available here (it will show some loading page but the PDF should download immediately): <a href="https://www.researchgate.net/profile/Won_Bin_Young/publication/315775073_In_Vivo_Excision_of_HIV-1_Provirus_by_saCas9_and_Multiplex_Single-Guide_RNAs_in_Animal_Models/links/58e644dfaca2727858cc2d67/In-Vivo-Excision-of-HIV-1-Provirus-by-saCas9-and-Multiplex-Single-Guide-RNAs-in-Animal-Models.pdf" rel="nofollow">https://www.researchgate.net/profile/Won_Bin_Young/publicati...</a>
Gene therapy (and CRISPR) is ultimately going to eliminate most if not every ailment/issue/genetic problem that we have, including aging and eye problems. Very thankful we are reaching the point where we can go into our cells and fix ourselves.
Just a reminder that MPEG LA has a CRISPR patent pool[1] and is willing to enforce it. It almost feels like satire.<p>[1] <a href="http://www.mpegla.com/main/pid/CRISPR/default.aspx" rel="nofollow">http://www.mpegla.com/main/pid/CRISPR/default.aspx</a>
From the abstract:<p><pre><code> Intravenously injected quadruplex sgRNAs/saCas9 AAV-DJ/8
excised HIV-1 proviral DNA and significantly reduced
viral RNA expression in several organs/tissues of Tg26
mice. In EcoHIV acutely infected mice, intravenously
injected quadruplex sgRNAs/saCas9 AAV-DJ/8 reduced
systemic EcoHIV infection, as determined by live
bioluminescence imaging.
</code></pre>
Can a mod change the title to reflect the findings? The original title is "In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models" and should be kept. That way maybe some people wouldn't just reflex-upvote and actually at least read the abstract...
In 2013 a very highly-placed Goldman Sachs exec, a friend of a friend, told me his AMFAR contacts predicted a 'genetics-based cure' in a 3-4 years instead of the 10 years they publicly projected. The rationale was donations would stop if people knew how close they were to a cure.
This, coupled with immunotherapy which boosts efficacy of our own defense mechanisms - for example, NK cell doping via LY49D/DAP12 - is quite exciting.<p>As someone else noted, THIS is what CRISPR was evolved to do. It was a DNA-based immune system used by bacteria to explicitly identify an invading virus via DNA, and to store a record of previously unknown virus for future reference. And, it was intended to function continually, in a living organism.
Craig Venter, the first genome sequenced and first promoter of shotgun sequencing, disses CRISPR. He claims its not reliable enough. Nor can you prevent from altering unexpected parts of the genome.
I dont know how much is not-invented-here and how accurate he is.
how did they refine crispr? Around this time last year we had<p>HIV overcomes CRISPR gene-editing attack
<a href="https://news.ycombinator.com/item?id=11453737" rel="nofollow">https://news.ycombinator.com/item?id=11453737</a>
Genomic responses in animal models do not mimic themselves in humans.<p>Great example is thalidiomide.<p><a href="https://www.quora.com/What-are-examples-of-drugs-that-showed-promising-results-in-clinical-lab-animal-trials-and-failed-miserably-in-human-trials" rel="nofollow">https://www.quora.com/What-are-examples-of-drugs-that-showed...</a><p><a href="https://walkerma.wordpress.com/2007/04/27/animal-models/" rel="nofollow">https://walkerma.wordpress.com/2007/04/27/animal-models/</a><p>I advise extreme caution and context on hyping this. Derek Lowe wrote a great post on this a few years ago that I still reference whenever I see HIV animal claims.<p><a href="http://blogs.sciencemag.org/pipeline/archives/2010/02/03/a_modest_literature_proposal" rel="nofollow">http://blogs.sciencemag.org/pipeline/archives/2010/02/03/a_m...</a>
Wow!<p>If this could be used to fight bacteria resistant to antibiotics then it is the start of something fantastic.<p>If it can be used to fight also diseases in animals (e.g.: foot & mouth disease in cattle) or plants (e.g.: citrus cancer) then it is even bigger.
As exciting as this is, we already know how people have used the decreased danger of HIV infection to become careless about other types of STIs. Given that our antibiotics are losing their ability to treat many common STIs, an HIV cure without some advancement in antibiotics will be swapping one epidemic for a series of others.<p>Edit: I wanted to add a couple sources for my claims above. Once people stopped believing raw sex was an existential threat they started going nuts, falsely believing that everything else can just be cleared up with a pill. Let me also say that my SO worked on the front lines of public health for almost a decade so I might have a unique perspective on this issue because of what she experienced as part of her job.<p><a href="http://www.cnn.com/2017/03/01/health/syphilis-newborns-partner/" rel="nofollow">http://www.cnn.com/2017/03/01/health/syphilis-newborns-partn...</a><p><a href="http://www.huffingtonpost.com/entry/std-rates-in-the-us-rise_us_5807bcf6e4b0b994d4c36002" rel="nofollow">http://www.huffingtonpost.com/entry/std-rates-in-the-us-rise...</a><p><a href="http://www.ocregister.com/2017/01/26/syphilis-up-412-gonorrhea-204-why-are-orange-county-std-rates-through-the-roof/" rel="nofollow">http://www.ocregister.com/2017/01/26/syphilis-up-412-gonorrh...</a>
It's awesome that this is possible. Right now HIV infections are basically a minor nuisance, provided you're taking the appropriate medication, but this bodes well for future treatment of viral infections.